Talk:Gene therapy/Archive 1

Archive 1

refactoring

Someone put the {{refactoring}} tag on the article 29 April 2005. I took that tag off today. Don't take that the wrong way -- I certainly want to encourage refactoring on this and other articles. --DavidCary 10:04, 6 January 2006 (UTC)

blahhh

Ashanti de Silva

I think this article should include info on Ashanti de Silva, who was the first gene therapy "success story". -Mjklin July 4, 2005 15:15 (UTC)

"supplement an effective mutant" isnt supplant the intended word? -Whistlingmonkey

I think a better idea would be to make a brief mention of this case and give him/her their own wiki. I'm afraid I'm not familiar with it, could you make mention of the link? Feel comfortable adding a mention yourself. Tyciol 14:08, 16 February 2006 (UTC)

Deletion of unnecessary redirects

Hey, could somebody delete the redirect pages "Gene Therapy" and "Human gene therapy" we really only need this one article - Zvesoulis 20:46, 26 Apr 2005 (UTC)

That sounds like a good idea. As far as I know, mods are the ones who delete things but you could mail them or something with this reason, they should comply. Tyciol 14:06, 16 February 2006 (UTC)

Human gene therapy and Gene Therapy would need to go through the Wikipedia:Redirects for deletion process. I think you would find little support for their deletion, though, unless you can argue that the existence of the redirects creates confusion or presents an undue burden to people searching Wikipedia for particular articles. User:Ceyockey (talk to me) 20:06, 4 March 2006 (UTC)

Requested Copyediting, Section: "Non-Viral Methods".

I corrected the apparent spelling errors and checked many of the scientific terms against Google, seems alright. The terminology is pretty hard to follow; Elaborate more and consider using less-specialized terms. --Avillia ^{(Avillia me!)} 04:30, 21 May 2006 (UTC)

Content taken from another source without credit

From the bad formating of the "probelems" list at the bottom it was clear it had been coppied from another source. A quick google brings up this, which is word-for-word identical. As a government website, is this in the public domain? Either way, the source should be credited. EAi 14:54, 6 May 2006 (UTC)

I hate to see things taken verbatim, but I do believe that publications of the US government are considerred in the public domain. However, I'm not sure if that blanket copyright coverage applies to organizations such as Oak Ridge National Laboratory, which are parts of the government managed by private sector companies. Nonetheless, I've added a reference to this material ... though I am not the source of the original input to the article. Regards, User:Ceyockey (talk to me) 13:16, 21 May 2006 (UTC)

Viral vectors

I suggest creating a new article on viral vectors using the material in this article as the foundation. Viral vectors are an important tool widely used in research as well as gene therapy. In that case there should just be a breif reference left in this article. Peter Znamenskiy 10:23, 21 Mayb 2006 (UTC)

I dunno about that, if we can find enough find enough articles to link to a vectors page - yeah maybe, but if you are going to move the viral vectors, move the non-viral ones too as they are becoming increasingly common in research. If it did happen I would call it Vectors in gene technology or something similar - and redirect viral vectors etc there. However what about pages like cationic lipids etc (if there isnt a page there the should be) as they have been created primarily for use in gene technology.) Viridae 13:00, 21 May 2006 (UTC)

Creation of a new article that would deal with viral vectors generically would be fine, but leaving only a minor reference here to that article would not be OK. The use of viral vectors in gene therapy is a major issue with regard to the public's perception and the medical side effects of gene therapy. Therefore, a full section should remain here that deals with the matter of viral vectors in the context of gene therapy (a revision or replacement of current content), aided by a {{main}} reference to the new article that deals with the subject of viral vectors specifically and thoroughly; where the new article ventures into 'uses in gene therapy', a reciprocal {{main}} would be in order referring to this article. User:Ceyockey (talk to me) 13:08, 21 May 2006 (UTC)

Okay, point taken. I've reread the section carefully and most of the material is in fact specific for gene therapy. I'll start on a general viral vector article and leave the material here. Peter Znamenskiy 00:17, 28 May 2006 (UTC)

Expert needed

I added the "expert" template tag today. I'm no expert myself, but there has been a great deal of work on this topic by professional bioethicists, and I think this article could benefit from it.

I added a narrative under 'social consideration' to offer a broader discussion of some of the possible social implications. --Oobasogie 22:17, 8 August 2006 (UTC)oobasogie 15:17, 8 Aug 2006

I added a section under "Human Enhancement" to help balance out the analysis a bit. Oobasogie 22:34, 14 August 2006 (UTC)

I've removed the seciton on positive reasoning and chimeras to streamline the information presented in the article. I"ve also removed the "significance" section since it is a bit speculative and its most important parts are presented in the "considerations" portion of the article. I've moved up the "process" and "when to make chnages" sections of the article to improve the flow of information. I've also tried to clean up the "process" section. Oobasogie 00:25, 25 August 2006 (UTC)

I cleaned up some of the prose in the opening paragraphs and removed the section on "Interference from Law" because it is no longer accurate. See http://www.latimes.com/technology/la-he-genetherapy28aug28,1,4998228.story?ctrack=1&cset=trueOobasogie 19:26, 6 September 2006 (UTC)

Latest News section

I just tagged the most recent research section for cleanup because as it is now, there are many missing links that could be used (like NIH should link to its article) and the references are in a flat format (and not even a correct one). The section text could be cleaned up to be more layman-friendly too. ju66l3r 17:25, 29 October 2006 (UTC)

Rough English

Lots of grammatical edits are needed in this article. The grammar is pretty rough, to be honest. For example, there are sentences of the type "The Subject Verbs the Predicate, however Another Sentence Is Running On Here." I changed one instance, but there's a lot more to be done, if any brave soul cares to edit the article. Spoxjox 18:27, 26 February 2007 (UTC)

The Process

The section titled, "The Process" should be rewritten in a third-person non-imperative point-of-view. —The preceding unsigned comment was added by Modernhiawatha (talk • contribs) .

Genetic engineering in fiction

There may be overlap with the article Genetic engineering in fiction. (SEWilco 05:54, 29 May 2005 (UTC))

With the examples listed, yes. Some non-fictional examples are needed.

Since genetic engineering of humans is generally off limits for various reasons, most literature on that subject may be fictional. Still, a lot of fictional literature (especially more recent literature) can be valuable for people wishing to get a grasp on a subject which may be rather opaque to the uninitiated. EthanL 10:40, 2 March 2006 (UTC)

I've added a reference to James Blish's The Seedling Stars. I think that a reference should also be added to Nancy Kress's work. There is a great deal to be added about the real-world ethics and politics, e.g. transhumanist views, bio-conservative views, etc. Metamagician3000 00:31, 3 January 2006 (UTC)

I'm not sure why we talk about "spiritual considerations". The issue raised is a metaphysical one about determinism versus free will that seems to have little to do with spirituality as such. I take it that the spiritual realm is a realm that transcends both body and mind, as in Hindu philosophy. Nothing like that is mentioned here, though there is a reference to the "soul". Metamagician3000 00:43, 3 January 2006 (UTC)

--Someone should consider putting in references about A Brave New World By Aldous Huxely- its entire plot and storylines revolves around the concepts of using genetic engineering and human conditioning to create a perfect society.--

Why does the blurb for When the Wind Blows read like it was copy-and-pasted from a book review? On another note, it would be nice if someone would add Maximum Ride to the list (it's loosely based on When the Wind Blows, and by the same author).

The entire list should be moved to Genetic engineering in fiction; that's what it's there for. Noclevername 06:44, 2 April 2007 (UTC)

That section has obviously gotten far too large, it should be merged with the already existing article Genetic engineering in fiction. I've added a merge tag. BTW I've also added a "Complete rewrite" template to the top of the article, this page needs to become a lot better to be up to any kind of Encyclopeadic standard. --Hibernian 00:05, 19 April 2007 (UTC)

siRNA more common and useful than oligodeoxynucleotides

I recommend that the section on oligodeoxynucleotides be reviewed and short interrupting RNA be discussed. Interruption at DNA level, from memory, is difficult and ineffective, where as transcriptional knock outs due to siRNA-mRNA interactions have been shown to be very efficient. siRNA techniques are very new, may have to search Pubmed reather than Google to confirm this. Cheers everyone.—The preceding unsigned comment was added by 60.224.23.251 (talk • contribs) .

The oligodeoxynucleotides papers were got from pubmed. I am at a loss as to how siRNA will be used - that seems to be a more severe modification than normal gene therapy, especially considering the effect of that sort of treatment to reduce total RNA as well as the target (or so I have heard). But then again, im in Phytopathology so what would I know :P Viridae^Talk 12:15, 10 March 2007 (UTC)

In the French gene therapy XSCID trial the three patients in which leukemia was reported have all been sucessfully treated. (no refs). The statement that they developed leukemia may be taken the wrong way. SCID is surely worse than leukemia?

80.177.16.166 23:53, 7 August 2007 (UTC)

Social considerations

This section and the following section (Metaphysical considerations) are criticisms, and should be designated as such. Also, the last paragraph in this section is unclear. Is the idea for governments to take control of genetic engineering their populations themselves or for taxpayer dollars to be paid to genetic engineering companies to engineer the populace? Either way, this is very controversial and should either be striken or ammended to include an opposing view. IntrepidDemise 08:34, 30 August 2007 (UTC)

Religious concerns

Hello, an anonymous editor raised the point about religious concerns here, which I then reworded here. I understand the point that the anon editor is trying to make, and I think it's a valid objection, though not one I personally share. It would help if there were a cited source for this, particularly because religion is such a touchy subject. --Kyoko 21:23, 4 November 2007 (UTC)

Edits

Organ transplantation is often considered to be the "original" form of gene therapy, that is, the insertion of foreign genes into a host to recover function. Many diseases which look to gene therapy for a potential cure can only be palliated by transplantation, sometimes of multiple organs.

removed since that just makes no sense to me, like saying buying a rebuilt engine is a form of engine repair (it's a form of AUTO repair, but not engine repair) - 165.247.224.196

I'd agree, because gene therapy would be modifying host cells. Organ transplant instead removes host cells and replaces them with alien cells (albeit, of the same species) so the effects are very much more hard to predict than modifying a single gene or two through gene therapy. It's more likely to be rejected and cause complications. Gene therapy is much safer, the only risk it has that organ transplant doesn't is the creation of side effects not priorly experienced by humans, which isn't really that horrible since you could probably predict a lot of them in the petri dish. Tyciol 14:06, 16 February 2006 (UTC)

Organ transplants are considered a form of gene therapy because it is replacing an organ with defective genes with an organ with functioning genes. Furthermore gene therapy is neither safer nor more effective than organ transplants at the moment, for the most part gene therapy is in its infancy, there have been many many unintended/unforseen consequences of gene therapy - for instance two children getting leukemia as a result of the retrovirus inserting itself near the promoter of the p53 tumor supressor gene effectively disrupting the promoter. Before extolling the virtues of Gene therapy (which is lookin fantastic but is by no means there yet) please do your research. I would suggest http://www.pubmed.gov . skorpion 03:39, 8 May 2006 (UTC)

However, if one is stabbed through the heart, it is not the genes the genes that are defective, but the heart itself. You could never, at least, with a mere organ transplant, hope to provide one with a heart that had genes that were not "defective"; that is to say, those that would allow the organ to be stabbed without consequences. By the greatest of technicalities, perhaps, but it is not for the sake of effective genes that transplants are performed. And what of the speculation of growing genetically identical organs, from clones? Is that gene therapy 76.105.251.120 (talk) 08:55, 8 January 2008 (UTC)

Sorry if i sound like an asshole but...

What kind of modifications is the gene therapy going to be able to do in a human body?

I was talking with a friend about this kind of medical technologies, and she told me that this therapy could be used in the future to treat transsexual patients, heal diseases like AIDS or cancer, and many things like that, is this true? And sorry if i sound like an ignorant but i want to get more data on this. Thanks. —Preceding unsigned comment added by 148.202.47.25 (talk) 17:17, 31 October 2007 (UTC)

I can't see how it would work on AIDS and to make a transexual change sex "naturally" you'd have to use some star trek method of beaming the right chromosomes into every single cell of thier body. However one possible use might be treating Cystic Fibrosis. People with this form mucus in thier lungs which prevents easy breathing and also lets bacteria grow. By 'sorting out' the genes in these cells the disease would be cured and eventually the mucus would clear away. That's a very basic description. 86.142.175.177 (talk) 15:59, 3 January 2008 (UTC)

1. 1. Trans-sexuality is not a disease, and the genetic basis for it is still unknown.
   2. Gene therapy is not useful for treating/curing infectious diseases like HIV. Adenoviruses (a common gene therapy vector) are currently being used in HIV Vaccine Trials -- this isn't gene therapy though, it's just injecting HIV material so the immune system can make antibodies to destroy any HIV in the body.
   3. Like the user above me states, well-understood genetic diseases like cystic fibrosis and thalassemia are first on the to-treat list. It really is as simple as adding three bases into one gene (for CF.) The problem is aiming for one very specific part of that gene -- and overcoming a huge amount of ethical and religious opponents. Danierrr (talk) 08:40, 13 January 2008 (UTC)

possible consequences?

I realize that if properly designed gene therapies based on viral delivery shouldn't effect reproductive cells and therefore won't enter the gene pool, but what's to stop a retrovirus from becoming endemic? An endemic retrovirus has a comparable and potentially worse effect than a retrovirus that can violate the Weismann barrier. Certainly giving everyone the same functional gene isn't that scary, but eliminating genetic diversity has had historically bad results. Vicarious 12:06, 10 March 2007 (UTC)

1. 1. The viruses used in gene therapy are incapable of causing disease. The gene(s) responsible for the disease they cause are ripped out, and we use the extra room for the "good genes" which can cure illnesses.
   2. There is some evidence for gene therapies crossing the Weismann barrier, but this is a good thing. If someone with cystic fibrosis was cured with gene therapy, if the Weismann barrier could be crossed, his/her children would be free of the disease as well. The treatment spreads from an individual to the population :)
   3. We are reducing genetic diversity -- by eliminating alleles responsible for causing disease. This is no different than eradicating smallpox and polio. Surely their genetic diversity was not missed! Danierrr (talk) 08:44, 13 January 2008 (UTC)

Current genetic engineering technologies on the web

There are numerous websites that are directly relevant to the topic of human genetic engineering, such as 23andme, decodeme, polonator (DIY pyrosequencing), DIY genetic engineering, and other websites (oww comes to mind) that are basically pathing the way for personal action when it comes to taking responsibility for your own genome. I think that this information should be incorporated into the article. Any suggestions? -- kanzure (talk) 01:38, 14 April 2008 (UTC)

Non-viral Delivery Methods

Due to certain problems using retroviruses there is a lot of research into non-viral delivery methods at the moment ie liposome mediated delivery

If I get the time I will do some research and rectify this, but in the meantime someone might want to do it for me. skorpion 03:38, 8 May 2006 (UTC)

I have started the addition of a section on non-viral vectors. Have thus far covered nked DNA and Oligodeoxynucleotides. Still to come is lipoplexes and polyplexes and possibly a mention of hybrid methods. I will link to my sources when I am finished in a few hours time. skorpion 07:32, 18 May 2006 (UTC)

Just had another read of what I werote and realised I need to dumb it down a bit. Will do that when I finsh the revision. skorpion 13:12, 18 May 2006 (UTC)

Ok, as of this point what I was going to add is finished. It is referenced as well. Can someone please read over it, check wording and selling mistakes. skorpion 06:36, 19 May 2006 (UTC)

Copy edit tag added skorpion 06:41, 19 May 2006 (UTC)

The last thing this article needs is dumbing down. Someone really needs to dumb it up, but I don't have time. ----Seans Potato Business 14:52, 31 May 2008 (UTC)

New developments

Found this on Salon, redirected from AP:

http://www.salon.com/wires/ap/scitech/2008/04/27/D90AECH00_vision_restored/index.html

It is related to the last mentioned case of gene therapy use in treating eyesight problems.

Hope someone can update, as I am not an experienced editor. —Preceding unsigned comment added by 217.129.111.158 (talk) 21:13, 27 April 2008 (UTC)

• I have added that yesterday under 2008 title. vcpk (talk) 15:47, 28 April 2008 (UTC)

• • Merged into the 2007 section, as it relates to the same trial. Neıl 龱 09:00, 3 June 2008 (UTC)

Sorry for putting this in what I expect is completely the wrong place. Simply wanted to alert you all to the fact that the development timeline was copied almost word for word from http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml 192.43.227.18 (talk) 01:33, 16 June 2008 (UTC)

Using Herpesviruses

Why hasn't anyone mentioned the use of herpesviruses in gene therapy?

Why don't you do it? skorpion 23:48, 16 May 2006 (UTC)

Please add I don't know about it, but I added section —Preceding unsigned comment added by 128.231.88.5 (talk) 16:26, 18 June 2008 (UTC)

Why is it prohibited?

Hi.

I saw this: "In the case of germ line gene therapy, germ cells, i.e., sperm or eggs, are modified by the introduction of functional genes, which are ordinarily integrated into their genomes. Therefore, the change due to therapy would be heritable and would be passed on to later generations. This new approach, theoretically, should be highly effective in counteracting genetic disorders. However, this option is prohibited for application in human beings, at least for the present, for a variety of technical and ethical reasons."

But it doesn't explain what those ethical reasons are! What are they? mike4ty4 (talk) 20:56, 13 December 2008 (UTC)

Ethical Considerations

This section uses presuppositions pertaining to Christianity, hindering its neutrality. —Preceding unsigned comment added by 220.246.244.63 (talk) 19:53, 22 November 2007 (UTC) The major problem that genetic engineering has is that once you mutate a particular gene with a substitution you don't know for sure what will happen. When chopping off a bit here and adding a bit there, how do you know you won't express a whole new set of problems? Perhaps a virus as virulent as hemorrhagic fever. There's never been a reservoir found for it. If a wrong signal is sent to a gene that has previously been coded and now there's an open slot in the DNA, who's to say that it won't open a whole new host of problems. Some that were never previously seen. We were designed with a certain concept at birth. It's like being exposed to a large amount of radiation: your DNA is changed forever, even through future generations.(Cobalt131 (talk) 04:24, 11 December 2007 (UTC))

You'd be amazed at how many genes are utterly worthless. It is not a computer program where every character is essential to the perfect design of an algorithm. Besides, that is what testing is for. I'd be more worried about accidentally modifying a chimp to be super strong, smart, and have overly developed ambitions of ruling the world.149.130.235.202 (talk) 18:45, 6 April 2008 (UTC)

Do not say that some genes are utterly worthless. We just do not know what they are for. (From: Some year 10 who thinks he knows it all.) —Preceding unsigned comment added by 58.106.83.128 (talk) 08:41, 10 April 2008 (UTC)

Genes are naturally altered all the time by reproduction, through random combination and mutations. If this kind of single gene tweaking disaster could happen, it would be quite ubiquitous, without even the need for engineering. What engineering could do is insert single genes which we know empirically to confer immunity to diseases like Aids. It seems that to induce a few proven beneficial genes would be safer than the alterations that happen by random chance through natural reproduction. JonatasM (talk) 04:54, 2 January 2009 (UTC)

Note - copying contents of Talk:Human genetic engineering as part of merge

Ok, I guess I should say something about it. I've made a slight change to the claim that germline engineering is "deemed inappropriate by most scientists." There is no reference to support this claim. The reference that does appear at the end of the article makes a considerably weaker claim: that it is not appropriate at present due largely to the unreliability of today's methods. If you want to claim that scientists don't find germline engineering appropriate at all, please at least give a citation that makes claims to that effect. Xezlec 16:57, 20 December 2006 (UTC)

I changed the number of chromosomes from 46 to 23. Humans have 23 distinct chromosomes - 2 pairs of each resulting in a total of 46 chromosomes. However, it is inaccurate to say we have 46 - that implies they are all different from one another (i.e. - no repeated genes / multiple alleles), while each pair of our 23 chromosomes contain 2 alleles of a given gene (one each). Had we 46 separate chromosomes every mutation would be dominant and that would lead to a great deal more disease and cancer. - Jonn

"Germline engineering would change genes in eggs, sperm, or very early embryos. This type of engineering is inheritable, meaning that the modified genes would appear not only in any children that resulted from the procedure, but in all succeeding generations. This application is by far the more consequential as it could open the door to the perpetual and irreversible alteration of the human species." - Alterations are neither irreversible nor perpetual: they can be reversed in the same way that they were induced. If there are no objections, I will remove this last phrase which seems incorrect. JonatasM (talk) 04:59, 2 January 2009 (UTC)

Axolotl and other genetic modifications

Regeneration of body parts and increased life-expanditure could also be possible for humans

Please include this in the article. Also, there are a wide array of other enhancements which may be helpful: for example I was thinking on elimination of fat-storage from foods (so that one cannot become fat anymore). Instead, the body can use muscle tissue for energy requirements if one is too active (and consumes more than his allowed 2000kcal; which would still be covered by a sufficient diet including a just large enough amount of goat milk, yoghurt or tofu and a single meal consisting of some vegetables, tofu (+only 1 portion of rice; 65gr of cooked rice) to fulfill these 2000kcal. Since pigs cannot make a fat layer neither (instead they have evolved to only "shivver" when its cold to keep warm), I guess this may be done in practice.

Another idea is the comparing the DNA of crairvoyants and modifying genes with other people in order to obtain extra-sensory perception, mentalism and/or other paranormal abilities (eg remote viewing, sending of spirits (see Egungun/Orisha, ... Given that the military has also poured money into psychic warriors, they may surely invest in these kind of ideas which are far more feasable. —Preceding unsigned comment added by 81.246.152.106 (talk) 16:53, 5 February 2009 (UTC)

Please look up whether some research has been done to the latter also, and perhaps include in article. Would be an important contribution.

Also add Robert Lanza's info from http://cosmiclog.msnbc.msn.com/archive/2009/02/12/1792488.aspx —Preceding unsigned comment added by 81.246.165.20 (talk) 08:02, 18 February 2009 (UTC)

Thanks, 81.246.137.55 (talk) 12:57, 13 December 2008 (UTC)

Also mention Armed Forces Institute of Regenerative Medicine (AFIRM) see http://www.popsci.com/military-aviation-space/article/2008-06/rebuilding-troops

finally I also heard about a snail that can use the power of the sun for energy (like solar panels, but for organisms). It was stated that the snail gets a gen by eating an algae while growing up, after which it can gather energy from the sun and needs less food. It was stated that this function could perhaps be built in with humns using genetic engineering. could thus reduce food requirements/production on earth —Preceding unsigned comment added by 81.246.162.121 (talk) 07:55, 6 April 2009 (UTC)

Body modifications

Why were all of the body modification explanations taken out. You've made this entire article about gene modification without once mentioning what possible changes could be introduced. People reading it who haven't read much sci-fi or literature work will understand that there is a general process that engineers go through.

Let me put it another way, we've written an article about engineering, and we've explained to the reader about the draft board and that an engineer can make a change, while leaving out most if not all insights into what kind of changes could be made in the design of people. So far you guys have lost the point of this article, which is not just to talk about how the engineering is done, but also about what kind of changes could be made. -- fieryfaith.

If you want to put in speculative ideas, cite respected futurists and scientists who have talked about it. No one wants to hear your own opinion. Djadvance (talk) 05:18, 13 April 2009 (UTC)

Book

Anyone who is interested in this sort of stuff should most definatly read the book Brave New World by Aldous Huxley. A classic and even though it was written in 1932, it makes clear connections to stuff happening in our world today. It is quite eerie. —Preceding unsigned comment added by 71.35.180.150 (talk) 05:04, 18 September 2007 (UTC)

Brave New World should be removed from this page. Since genetic engineering didn't exist when Huxley wrote it there is no depictions of genetic engineering in the novel. The characters in the novel use environmental and psychological methods to modify children not genetic engineering. —Preceding unsigned comment added by 71.192.147.94 (talk) 17:36, 23 April 2009 (UTC)

Aubrey de Grey

Can Aubrey de Grey and SENS be mentioned ? —Preceding unsigned comment added by 81.246.169.78 (talk) 12:16, 28 April 2009 (UTC)

benefits

can it be mentioned that monogenetic deviations (such as diabetes, muscle dystrophy and multiple sclerosis) can propbably be cured soon with gene therapy —Preceding unsigned comment added by 91.182.203.223 (talk) 16:30, 2 July 2009 (UTC)

Puff piece

I think the HUGE hype and failure of gene therapy ought fairly to be discussed here and the death of a perfectly healthy young man due to gene therapy --and the both the lead up to and aftermath of that incident--deserves more than a one-line mention. —Preceding unsigned comment added by Eperotao (talk • contribs) 05:56, 5 June 2009 (UTC)

added some information to the Adenovirus section to address this concern --JimHu (talk) 21:29, 27 June 2009 (UTC)

The young man you are talking about was named Jesse Gelsinger (there’s an entry for him in Wikipedia). He suffered from ornithine transcarbamylase deficiency meaning that he was not perfectly healthy. Before enrolling in the clinical trial of the U of Penn, he treated his disease with a special diet and medications. At the time of gene therapy treatment he was symptomatic with elevated serum ammonia, which is a condition that can be fatal. An investigation by the FDA found that his death was due to organ failure triggered by an immune response to the gene therapy product (a replication-defective adenovirus). However, that doesn't provide the complete story. During the investigation, the PI of the trial Jim Wilson was found to have made very poor decisions regarding the care of his patients. First, he ignored the exclusion criteria which precluded patients who were symptomatic (i.e., elevated serum ammonia). In other words, according to the exclusion criteria set up by Dr. Wilson, Mr. Gelsinger should never have been enrolled in the trial let alone treated. Dr. Wilson also failed to report severe adverse events observed in patients treated previously and failed to report deaths of primates in preclinical studies. As a result of the findings of the investigation, Dr. Wilson has been barred from conducting gene therapy trials. The death of Mr. Gelsinger was a tragedy but I’m not sure we are ready to throw in the towel and say gene therapy is a huge failure. If I had a child with SCID I would without hesitation enroll my child in a gene therapy trial. That’s even with the knowledge of the increased risk of the child developing leukemia-like symptoms as a consequence of the gene therapy. For context I recommend you read some of the early studies of various chemotherapeutic agents that are used regularly in the clinic today. These agents are extremely toxic and resulted in the deaths of many patients during the testing phase. Yet we have no objection to their use. Gene therapy still provides great promise to help patients live with many different metabolic disorders and diseases but our enthusiasm must be tempered by the potential risk.(Fgrnis (talk) 18:43, 10 July 2009 (UTC))

stem cell gene therapy

You will have to excuse my ignorance, but I wonder in wich vector/method, stem cell mediated gene therapy would fit Thank you!!! —Preceding unsigned comment added by 114.73.49.239 (talk) 07:11, 28 November 2009 (UTC)

HELP

Explain how gene therapy would be used to treat SCID(severe combined immunodeficiency syndrome). —Preceding unsigned comment added by 74.131.21.39 (talk) 16:14, 20 October 2008 (UTC)

This is not a homework help site, do some research yourself and read this article for any info. Tory88 (talk) 14:23, 5 December 2009 (UTC)

Problems and Ethics

This is just a small matter, but under the heading Problems and Ethics there is no mention of ethics, just mentions of problems encountered. Should this be changed? 63.165.44.97 (talk) 23:23, 18 February 2010 (UTC)

Probably, with any big controversies. MichaelExe (talk) 03:07, 24 February 2010 (UTC)

Metaphysical considerations

I think the subsection metaphysical considerations should be removed. Neuroscience and psychology are the areas of science dealing with personality, not human genetic engineering. —The preceding unsigned comment was added by 129.241.127.122 (talk • contribs) .

Rewrite

This article is in desperate need of a rewrite. There is far too much speculation and theorizing, not to mention directly asking the reader if it is ethical. JDub90 18:39, 15 May 2007 (UTC)

Well 3 years later, can't say much has changed. This page is really embarrassing. The editor responsible for it should be banned from Wikipedia until he/she graduates from middle school. 92.78.110.148 (talk) 20:30, 15 March 2010 (UTC)

Incorrect definition

The definition currently on this page ("Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one.") is very narrow-minded and betrays the ignorance of the author. It also contradicts the examples given in the text. Replacement of a mutant allele by a normal one is a very specific field and, arguably, the most technically difficult route of gene therapy. Gene therapy could be administered by expressing a gene product which is missing from a cell (without replacing or correcting the broken gene)or even introducing and expressing an artificial gene which achieves a therapeutic result, but does not necessarily resemble a normal gene (e.g. gene therapy of cancer with a vector which codes for molecules that are detrimental to the cancerous cells). —Preceding unsigned comment added by 72.88.65.200 (talk) 02:49, 24 February 2010 (UTC)

Also, I would just like to add that this article is probably the worst-sourced, worst-written article I've ever encountered in Wikipedia. Many sentences don't make sense, the citations are incorrectly labeled and half of the information isn't even cited. In one instance, it says "A fire broke out in human subjects." Some brave person should consider cleaning this article up. (66.253.161.201 (talk) 23:51, 12 April 2010 (UTC))

genes play role in natural death (apoptosis)

True or false:

It is "written" in our genes when we will die. Genes control the life cycle of the cell. So, if apoptosis, or programmed "cell death" could be controlled through gene therapy or genetic manipulation, human-kind can essentially attain "biological immortality" (granted other diseases do not lead to death) within the next century as genetic research advances. Obviously the matter is much more complex because more than one gene is involved, as well as creating a balance so that cells do not reproduce uncontrollably, for example. But, it can be done. —Preceding unsigned comment added by Mgaribaldi117 (talk • contribs) 22:18, 13 September 2010 (UTC)

Missing page in first external link

The first external link links to a 404 page. Should a cached verson be found or just deleted? --38.116.202.9 (talk) 13:31, 16 November 2010 (UTC)

This page sucks

This article sucks so much it should just be deleted. It sounds like someone in middle school wrote it. It's made me embarrassed to use Wikipedia. —Preceding unsigned comment added by 92.78.110.148 (talk) 20:33, 15 March 2010 (UTC)

Then do something about it, find sourced material, don't just bitch.'''Aryeonos''' (talk) 05:35, 23 December 2010 (UTC)

Actually, this page is a goldmine of useful information. For instance, did you know that "the protagonist and several other characters in the Halo universe known as Spartans have all gone through comprehensive genetic augmentation, making them almost super human. The augmentations vary from Muscular Enhancement Injections to a Catalytic Thyroid Implant. The effects include nearly unbreakable bones, increased reflexes and increased muscle tissue with increased density. The SPARTAN-III program also included a mutagen that increased aggression and injury tolerance." Wow! — Preceding unsigned comment added by 75.93.51.158 (talk) 04:22, 17 November 2011 (UTC)

Subarticles needed

This page is in desperate need of subarticles. The main page is far too long and too technical for a casual reader to be able to understand what is really going on in gene therapy; particularly if they are looking for human gene therapy, which re-directs here. I suggest moving the bulk of "methods of gene therapy" to a subarticle, and trimming back here substantially. — Preceding unsigned comment added by Thatbiotechguy (talk • contribs) 15:48, 29 December 2011 (UTC)

Recent revisions

Hello peeps. I am trying to revise / rewrite this article to update it given recent developments in the field. It is outdated, factually incorrect in many places, and contains too much detail of obscure topics for a general article. Therefore I recommend several revisions by adding subarticles (see below), making the intro better for a lay audience, and updating it to include more recent successes and failures in the field. I welcome your comments/feedback here or on my userpage (thatbiotechguy). Thanks. — Preceding unsigned comment added by Thatbiotechguy (talk • contribs) 18:06, 29 December 2011 (UTC)

Beginner questions

• Is gene therapy supposed to affect the whole body, or is it supposed to only affect those cells where it is neccessary (e.g. some gene defect causing gastrointestinal problems only requires gene therapy in the gastrointestinal tract)?
• Do cells treated with gene therapy participate in mitosis? Will copies of treated cells have the same treated DNA?
  • Is it required to constantly get gene therapy to keep the desired result, or is the purpose that once cells are treated the therapy is self-sustaining?
  • Is there a "combat"/"balance" between non-treated cells and treated cells? Are genetically-treated bodies technically Chimera (genetics)?
  • Does gene therapy have the target to completely transform the DNA of the patient?

--Abdull (talk) 15:38, 2 November 2012 (UTC)

Major Revision Required

The tag that this article needs be rewritten from scratch have been here for 4 years now. Reading through this article, it is indeed absolutely terrible and written more like an opinion piece rather than an encyclopedia. I will try to put some time toward rewriting this article. Are there any active editors that wish to assist in rewriting? BlackHades (talk) 21:34, 16 July 2013 (UTC)

I'm familiar with genetic engineering, so I can offer some help (though no guarantees for the long term). The articles on genetic engineering and gene therapy are much better maintained than this one and are likely good places to get information. Arc de Ciel (talk) 06:17, 19 July 2013 (UTC)

Thanks for the assistance. I will look through genetic engineering and gene therapy. Are there sources that specifically centers around this topic that you'd recommend? BlackHades (talk) 10:31, 19 July 2013 (UTC)

Unfortunately, I don't know the literature on this field that well, just the general background. :-) So I know how most of the techniques work, who some of the experts are on a couple of the topics, and I can point out if a particular source doesn't seem to understand the science. But I would say that it's too broad a topic (and not enough is known) for it to be covered in the scientific literature as a single unit.

That said, these reviews might be useful - please let me know if I can explain anything for you. I've just added them to a couple of articles, so you may come across them in Wikipedia as well. :-) I can also recommend more sources on any specific topics of interest.

• Esvelt, KM.; Wang, HH. (2013). "Genome-scale engineering for systems and synthetic biology". Mol Syst Biol. 9: 641. doi:10.1038/msb.2012.66. PMID 23340847. {{cite journal}}: Cite has empty unknown parameter: |month= (help)
• Tan, WS.; Carlson, DF.; Walton, MW.; Fahrenkrug, SC.; Hackett, PB. (2012). "Precision editing of large animal genomes". Adv Genet. 80: 37–97. doi:10.1016/B978-0-12-404742-6.00002-8. PMID 23084873. {{cite journal}}: Cite has empty unknown parameter: |month= (help)

General comments that you may find useful

Gene therapy is the field that's furthest along. Related articles of interest include vectors in gene therapy (aka delivery methods) and genome editing. There are also techniques such as SCNT (where the entire nucleus is replaced - this is how Dolly was cloned), but I hesitate to consider those genetic engineering. Another field that comes to mind is regenerative medicine, although that article isn't nearly comprehensive. This (and the related field of tissue engineering) mainly focus on applications such as the growth of organs for transplant, starting from stem cells. However, it doesn't currently use much genetic engineering; the closest it gets is genetic reprogramming. As far as I'm aware, the use of genetic engineering to give humans new traits (as opposed to prevention or cures) is mostly speculative, although the techniques should apply in the same way. Synthetic biology is the closest (in that it attempts to devise new traits), but it's still just beginning and is extremely far from any application in humans. I imagine that most of the descriptions of technology will be like this (not yet applicable to humans but could be in the future), other than that which applies to human cells in culture. The scope of the article is uncertain to me. Most of the sources that use "human genetic engineering" as a viable description discuss ethics rather than science. (Of course, the ethics is important as well, but my familiarity is with the science.) There is a lot of scientific detail that can be discussed, but as I mentioned above, the science itself doesn't usually use such a broad categorization. The science is moving quickly. An article written in 2012 may have a significantly different background context from one written in 2011. Modification is easiest at the zygote or blastocyst stage. There is a bit of information on how this is done at transgenic mouse. (It's not done in humans, of course.) There are a lot of people who use language such as "the next stage in human evolution." (This is something I removed from the article.) I think I also saw it in a couple of respectable sources when I was editing this page, but it's at the least imprecise/poor wording and (at least in my opinion) should be attributed if included. It is valid under a broader definition where "evolution" simply means "change over time," but it implies some mechanism of progress or goal-directedness ("stages") - so it is somewhat misleading when applied to biological evolution, which doesn't have these properties.

I won't be available much for at least the next few days, but please let me know if you have any questions. :-) If it's more than a week, please feel free to remind me on my talk page (since the message will drop off my watchlist). Arc de Ciel (talk) 10:35, 20 July 2013 (UTC)

Add external link

I would like to add an external link: Gene Therapy Net (www.genetherapynet.com). Gene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials—The preceding unsigned comment was added by 131.224.251.103 (talk • contribs) on 12:32, 31 January 2011‎. Jytdog (talk) 02:23, 29 December 2013 (UTC)

A type of Gene therapy

The sentence here says, "Antisense therapy is not strictly a form of gene therapy, but is a related, genetically mediated therapy." Isn't antisense therapy really a type of gene therapy? --سمرقندی (Samarqandi) (talk) 01:16, 20 January 2014 (UTC)

not really - antisense doesn't seek to alter the genome, but instead acts more like a typical small molecule drug, to interfere with the function of a biomolecule downstream of the genome. I edited the article to address this confusion. Jytdog (talk) 18:10, 20 January 2014 (UTC)

Thanks for reply. I do not know any peer review journal to mention it about antisense therapy, there are many journal articles and books that mention the term - Antisense gene therapy - I am not insisting but maybe we need more editing there or some reference. --سمرقندی (Samarqandi) (talk) 06:26, 21 January 2014 (UTC)

Intro: Gene therapy was first conceptualized in 1972; Development of gene therapy technology: 1970s and earlier

I've noticed this page doesn't speak of anything prior to 1972 and wonder if the addition of an article by E.L. Tatum in 1966 may be beneficial. In it, he discusses the potential uses of genetic engineering with quite a bit of depth for the time frame.

The article is: Tatum EL. Molecular biology, nucleic acids, and the future of medicine. Perspectives in Biology and Medicine. 1966;10(1):19-32.

and a reprint can be found at: http://www.ctt-journal.com/index.php?id=7&uid=140&code=DNL&backPID=7&no_cache=1&rtekeep=1

KahMisz (talk) 01:42, 26 April 2014 (UTC)

Hurdle: viral gene therapy regaining ability to cause disease?

In the section describing potential hurdles/challenges for gene therapy, I found the claim "The virus may regain its ability to cause disease." I am not well versed in the literature on this topic, but feel that it would not be possible for a viral vector to regain its ability to cause disease. It may be possible for it to maintain this ability through an improper manufacturing technique, which should be sufficiently addressed through cGMP practice and modern regulatory approval processes, but to "regain its ability" suggests that it correctly lost all viral genes and then (magically?) regained them. I see no possibility of this and have thus deleted the aforementioned statement. I call on anyone who believes that regain of function is possible to substantiate their belief with one or more references before posting such an unlikely claim to Wikipedia. Thanks! Cheddar3210 (talk) 02:25, 3 June 2015 (UTC)

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Proposal to merge in Pharmacological gene therapy

The following discussion is closed. Please do not modify it. Subsequent comments should be made in a new section. A summary of the conclusions reached follows.

The result of this discussion was to merge Pharmacological gene therapy in to this page Klbrain (talk) 22:55, 29 December 2015 (UTC)

---

I propose that Pharmacological gene therapy be merged into Gene therapy. I think that Pharmacological gene therapy article is almost a synonym, and can easily be explained in the context of Gene therapy. The Pharmacological gene therapy is so small, and has so little cited content, that it will not significantly impact the structure of the Gene therapy page. Klbrain (talk) 21:03, 30 August 2015 (UTC)

Seems a good idea to me. I'd vote for that.SylviaStanley (talk) 23:02, 30 August 2015 (UTC)

I agree they both seem linked to me. 88.104.140.187 (talk) 19:36, 17 September 2015 (UTC)

• Support. Lfstevens (talk) 07:38, 19 September 2015 (UTC)

The discussion above is closed. Please do not modify it. Subsequent comments should be made on the appropriate discussion page. No further edits should be made to this discussion.

Assessment comment

The comment(s) below were originally left at Talk:Gene therapy/Comments, and are posted here for posterity. Following several discussions in past years, these subpages are now deprecated. The comments may be irrelevant or outdated; if so, please feel free to remove this section.

Rated "top" due to medical interest and media coverage. - tameeria 23:50, 18 February 2007 (UTC)

Last edited at 23:50, 18 February 2007 (UTC). Substituted at 14:50, 1 May 2016 (UTC)

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Hello! Great page on Gene Therapy. It is very interesting, full of information and easy to read and understand. Thought I would leave a few suggestions for improvement if you decide to go back and do some editing. The background does a good job at introducing the topic, however as a suggestion to draw readers in, I think it might add to the article to include a little bit more information on the approaches mentioned in the background. In your section on hurdles involved with gene therapy, not all of the bulleted points have citations to go along with them. Even if the point came from a source that was previously cited, for the reader to be able to learn more, it may be helpful to have a citation for every one. While on the subject of sources, the links in your citations section work great! However, source #92 leads me to a source that is not in English. Consider changing the citation to an English version of that page. I enjoy that pop culture was involved, however as a reader, I wanted to know more about how gene therapy was involved in and interpreted in Stargate, I am Legend and "Will Gene Therapy Change the Human Race?" Consider adding more info on these connections if it is available. Have a wonderful day! HappyForestCreature (talk) 23:38, 16 January 2017 (UTC)

Aptamers

User:Cambamslamjam here and here you have added the following: (This is the last version)

Aptamers are non-viral targeting elements that are single-stranded nucleic acids with high specificity towards their associated targets. This class of bio-affinity elements is molecularly engineered to recognize and bind selectivity to particular cells, proteins and tissue in vivo with minimal damage to surrounding cells . Aptamers have risen as a class of chemically and thermally stable targeting compounds due to their phosphodiester bonds and a significant restorative potential for their biophysical and biochemical attributes of low immunogenicity, high efficiency, and biodegradability .

Aptamers that are oligonucleotides can either be single-stranded deoxyribonucleic acids (ssDNA) or ribonucleic acids (ssRNA) . They are generally utilized as cell targeting compounds in clinical diagnostics because of their high selectivity, which comes from the 3-D structure of the aptamer. The aptamers unique conformation binds to targets with a dissociation constant in the range of pico-molar to nano-molar by means of hydrogen bonding, Van der Waal connections, and electrostatic interactions . The interaction between an aptamer and its target tissue depends on the 3-D structure of the aptamer, which enables the agents to recognize minor distinctions between binding sites within a molecule of interest. For instance, the pegaptanib aptamer is an FDA-affirmed aptamer-based medication to target vascular endothelial growth factor (VEGF) for the treatment of age-related macular degeneration. The treatment shows high efficacy because of the interaction between the heparin binding domain (HBD) and the aptamer that determines the affinity in the VEGF165–aptamer complex . Pegaptanib is an example of an aptamer that is utilized as the aptamer-drug conjugate .

An in vitro approach to generating aptamers that have a specific target ligand(s) is Systematic Evolution of Ligands by Exponential enrichment (SELEX). The method of SELEX involves the synthesis of long oligonucleotides libraries with random sequences. These library sequences are then exposed to a target cell, tissue, or protein. The oligonucleotides that do not bind to the target are removed and the remaining sequences are then amplified . This methodology allows for the construction of aptamers that have a large range of target specificity for the desired substrate. Aptamers can be used as drug carriers and as biological drugs . An example of an aptamer used as a drug itself is the NS2-specific aptamer . The aptamer showed to have efficiency in preventing viral RNA replication, which can lead to anti-hepatitis C infection. It has also been suggested that pharmaceutical carrier aptamers can enhance the efficacy of antitumor agents on a variety of cancers . Overall, there are few aptamer-based therapies in clinical trials, including the nucleolin-particular AS1411 aptamer for treating acute myeloid leukemia and renal cell carcinoma, and the NU172 aptamer for the target of thrombin particles to treat anticoagulation in coronary illness..

The current hurdle for general non-viral gene therapy is to optimize the bioavailability of the agents at their targets while limiting systemic cytotoxicity . One of the limitations to aptamer-mediated targeted delivery systems is that there are only a few commercialized aptamer-based drugs . This is because it is common that aptamer delivery systems fail during clinical trials such as the AS1411 DNA aptamer, which failed to treat renal cell carcinoma within pre-clinical requirements . There are biochemical challenges that inhibit effective aptamer-mediated targeted delivery in vivo. The targets for aptamers frequently reside in interstitial fluids and are often internal surface protein receptors or biomarkers, which means it is vital that the aptamers are stable compounds that can withstand the in vivo microenvironment. The stability of an aptamer is challenged by having a short circulating half-life due to rapid biodistribution of the aptamers from the blood plasma to tissue, the electrostatic repulsion of the negatively charged cell membrane and negatively charged aptamers, and endonuclease degradation . The kidney also complicates the aptamer dosage to targeted sites because of rapid renal excretion . Lastly, there is the chance that aptamers can fuse into the genome to express foreign proteins that can potentially lead to cancer .

References

Thanks for wanting to improve Wikipedia! There are several issues with this content that need fixing where ever it ends up but the first question is, why do you believe that aptamers are a form of gene therapy at all? Once we solve that we can discuss the other stuff. Jytdog (talk) 03:30, 30 March 2017 (UTC)

lovely but sourcing is all unclear

per the header...

also it is not at all clear to me that this is talking about actual gene therapy. a lot of these appear to be more just general with regard to transfection per se.

Advantages and disadvantages of viral and non-viral vectors in gene therapy

Vector	Adventages	Disadventages
Viral
Retrovirus	They are very well studied and effective in the process of transferring DNA into the cells. Long-term expression Low probability of recombination	Small insert size (up to 8 kb DNA) Transfection only those cells that are in active dividing Risk of incision mutagenesis
Adenovirus	Transfection of replicating and non-reactive cells High expression of inserted genes Ideal for pulmonary tissue Possibility of aerosol application Rare intergration in the host genome	Low insert size (7–8 kb DNA) Significant risk of inflammatory response Short-term gene expression The vector is lost during the cell division Risk of recombination in the host Expression of other proteins
Adeno– associated virus	Long-term expression and its high safety	Small size of the insert (7–8 kb DNA) Non-productive infection without helper virus
Herpes virus	Transfection of replicating and non-reactive cells Especially good for nervous tissue Rare intergration into the host genome Insert size up to about 30 kb	Risk of recombination in the host Short-term gene expression
Non-viral
Liposomes	They do not carry viral genes, so they do not cause disease	When transferring gene, it is less effective than viruses Problems with vector size, which limits the amount of transferred DNA
Lipoplexes	They have no immunogenic activity, but are more suitable for transfer than viruses	The size limits the amount of transferred DNA
"Naked" DNA	Very useful for the construction of vaccine Useful in special cases	Inefficient in gene transfer In most tissues it is unstable
Microinjecting	Safety, precision and ease of transfer	Possible irregular proliferation in tissues It should re-injection
Biolistics	Safe transfer High success of transfer of genetic material to different tissues	Limited gene transfer efficiency Insufficient integration of injected DNA
Receptor-mediating endocytosis	A light infusion through the liver through the bile duct, where they are taken by hepatocytes, and transported to proliferating cells of hematopoietic tissue	Not designed to integrate the transferred genes The protein-DNA complex is unstable in the serum

References

-- Jytdog (talk) 21:19, 31 August 2017 (UTC)

Why did you delete the good and correct table in this article? It is quite constructive summary of problem treated.~ Evo-taxis (talk) —Preceding undated comment added 22:10, 31 August 2017 (UTC)

In addition, in gene therapy, vectors are particularly important. The success of the therapy depends on the correct choice. Therefore, this table is need of this article. Evo-taxis (talk) 22:32, 31 August 2017 (UTC)

First - again welcome to Wikipedia!! Am so glad you want to improve things.

Second - I moved this here so we could discuss it. You made a big change, and it is not clear to me if it is a good one. So we discuss things. No big deal.

Specifically here... Sourcing is unclear and I am not sure about that the content is about gene therapy per se. Also... Why did you decide to do this with a table instead of discussing things narratively? There is stuff about gene therapy per se that doesn't fit nicely in a table... Jytdog (talk) 22:35, 31 August 2017 (UTC)

I tried in "talk", but it did not go. Evo-taxis (talk) 23:58, 31 August 2017 (UTC)

You are talking! That is what this section is. Would you please say why you decided to create a table, instead of doing this narratively? thx Jytdog (talk) 00:24, 1 September 2017 (UTC)

"treatment"

So almost none of these are medical treatment but rather clinical research. The table makes it appear that gene therapy is way more ... realized than it is. Individual entries are also not sourced...

tables have their uses in WP but this one is not so much in my view.

The first diseases treated with gene therapy

• Melanoma, neuroblastoma, brain malignoma, cance of head, and neck, pulmo, liver, kidney ovary, breast cancer, colon, prostata, mesothelioma, leukemia, lymphoma, multiple myeloma.
  
• SCID = Severe Combined Immunodeficiency

References

-- Jytdog (talk) 17:38, 2 September 2017 (UTC)

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Human genetic engineering

This is stated on the article: "Genetic engineering could be used to cure diseases, but also to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence."

Should it be: Gene therapies could be used to cure diseases, but can also be misused to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence.

? Else, it seems out of place on this article (because we already have the human genetic engineering article now) and needs removal. --Genetics4good (talk) 16:49, 26 October 2020 (UTC)

Is mRNA "gene therapy"?

There is some recent discussion that tries to falsify the idea, but they seem to be using a pretty silly distinction. RS describe mRNA as a non-viral vector under "gene therapy", pretty easy to find. Reviews like that should be cited here.Maneesh (talk) 08:26, 1 September 2021 (UTC)

In general, no nuclear entry -> not a gene therapy. mRNA vaccines just provide brief, volatile instructions to provide a certain protein once. Gene therapies properly so called need to replace a protein for life, e.g. SMN1 with zolgensma. For that reason, they need to incorporate the gene into the patient's genome. This is not the case for mRNA vaccines. Ari T. Benchaim (talk) 00:55, 12 September 2021 (UTC)

no nuclear entry -> not a gene therapy Can you provide a source? Because I'm seeing academic sources that say that e.g. mRNA vaccines are forms of gene therapy sensu largo.[1]. I'm also seeing articles that variously count ASO-mediated RNAs as a gene therapy or as not a gene therapy. There clearly is a debate in the academic world about the exact definition of "gene therapy".

One definition I've come across and like very much is: gene therapy = nucleic acids used as therapeutics. What would be your take on it? — kashmīrī ^TALK 16:51, 25 November 2021 (UTC)

Our lead says Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material.. This does not include mRNA vaccines. Like GMO gene therapy has a range of different meanings depending on the time and place (and in some cases motivation) of people. Aircorn (talk) 21:51, 25 November 2021 (UTC)

The lead sentence was changed somewhere in 2020 and comes almost verbatim from the cited review article from 2001, which is very old, especially when it concerns a heavily evolving field such as this one [2]. In 2001, many of the techniques that were later developed under the umbrella of gene therapy were not known, which lead to this more restricted definition. The sentence that existed up until 2020 better reflects the use of gene therapy in literature, as it said: "Gene therapy ... focuses on the utilization of the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease". There are many examples of the use of this word that involve mRNA and no nuclear entry, for example [3] which even directly cites the lack of need for nuclear entry as an advantage. Other examples are [4] and [5]. 2A02:1811:D13:4A00:CB88:D0AC:5175:CD4B (talk) 17:28, 29 November 2021 (UTC)

Further research suggest that as far as regualtion goes in the USA it is not and in Europe it is. This pretty much mirrors thinking along these lines with CRISPR and other GM regulations.[6] Aircorn (talk) 22:06, 25 November 2021 (UTC)

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Gene therapy

Detail information about gene therapy and recent advances in it. 59.103.251.24 (talk) 07:49, 6 March 2023 (UTC)

DNA vaccines and regulation

I removed a sentence from the lead using EMA regulation (pre-covid) to talk about the definitions of gene therapy. Some relevant sources on this are. and .

I think a fair summary would be:

"In Europe, DNA vaccines are regulated by vaccine regulation rather than the regulation for gene therapy medicinal products. When similar vectors are used compliance with gene transfer regulation is encouraged."

We may well want to remove "or through altering the biological properties of living cells." as well... in the critique of the FDA definition in the breadth of definition section, the author argues that this definition is broad enough to include any medication. Talpedia 19:27, 17 June 2023 (UTC)

My intention was to offer a single widely accepted definition of a gene therapy, by the latter term meaning a medical technology, i.e., a class of medicinal products. I have no strong opinion about vaccines being called gene therapy. Let me just note that mRNA-based vaccines indeed aim to modify gene expression by inserting nucleotides into cells; however, they are used for prophylactic, not for therapeutic purposes, so the term therapy may sometimes be questioned (even as EMA definition allows the use of gene therapy for products used for diagnostics and prophylactics). I'm open to discussion. — kashmīrī ^TALK 20:37, 18 June 2023 (UTC)

I think a general definition would be good. I'd prefer to get it from textbooks or something along the lines of "the philosophy of gene therapy". I found at least one pretty good textbook on the topic before. Perhaps the "prophylactic" versus "therapeutic" argument is plausible... on the other hand I've seen a lot of contortions going on around this topic (e.g. rna doesn't count as gene therapy because it doesn't last long enough, gene therapy needs to modify the genome, gene therapy needs to cause long lasting changes). It's quite interesting that mRNA seem to mostly come from vaccine research and are moving into "gene therapy proper", while the AZ vaccine feels like "mainstream gene therapy".

I looked at ISBN:9783030413323 a bit before. I guess there's value here in that the source synthesise a few different definitions together:

In a simple way, gene therapy could be defined as a set of strategies modifying gene expression or correcting mutant/defective genes, which involves the administration nucleic acids - DNA or RNA - to cells. However, more elaborate and complete definitions for gene therapy can be found, especially the ones produced by regulatory agencies. According to the European Medicines Agency (EMA) and to the European Union (EU) directive 2001/83/EC, a gene therapy product consists on a biological medicinal product, which has the following characteristics [1]: (a) it contains an active substance, which includes or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding, or deleting a genetic sequence; (b) its therapeutic, prophylactic, or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains or to the product of the genetic expression of this sequence. Gene therapy medicinal products do not include vaccines against infectious diseases. For the US Food and Drug Administration (FDA), gene therapy is the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use [2].

This references the EMA definition... so there is an argument for including it, it also starts off with a definition of along the lines of using DNA or RNA to modify gene expression. Interstingly the EMA definition includes prophylactic uses... Talpedia 21:14, 18 June 2023 (UTC)

For some general discussion of the definition see the "Breadth of definition" section. I basically think there is no definition, and the FDA actively avoided a concrete definition preferring to pick and choose what counts as a gene therapy. DNA vaccine researchers actively define their work as gene therapy and publish in gene therapy journals. The whole "vaccines are not gene therapy" thing is a COVID-period construction to try to create a boundary around the COVID vaccines both in terms of fighting Fear, uncertainty, and doubt and narratives supporting incorrect statements about vaccines modifying genes.

Fact checkers gripped with "vaccine compliance zeal" have reached for regulatory definitions to create distinctions that don't actually exist. Talpedia

References