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In a First, US Doctors Use Crispr Tool to Treat Patient with Genetic Disorder
- tyingq 7y agoAssuming they use CRISPR outside the body, on blood, then infuse the blood back in... Are there any credible risks that something unexpected happens?
- AndrewDucker 7y agoYup: "During a routine data run, they unexpectedly found foreign, non-bovine DNA that had bound itself to the animal’s genetic sequence during the edits—specifically, genes from the lab material" https://newfoodeconomy.org/fda-gene-edited-cattle-antibiotic-resistant-crispr-dna/ https://newfoodeconomy.org/fda-gene-edited-cattle-antibiotic...
- justinjlynn 7y agoWhat's the probability that the sequencing sample was contaminated and that the sequence result doesn't represent the animal in question? Has that been ruled out? Obviously such integration errors occur in reality. Though, in therapy I'd expect the rates to be quite low. It'd be interesting to find out how often it happens in these applications.
- aae42 7y agosuperheros?
- ben_w 7y agoWell, the Milkybar Kid is one of the few superheroes whose origin story has not been done to death in the endless reboots that afflicts the genre, so that could work as a good story…
- mbreese 7y agoIntegration errors aren’t the main concern with treating patients - it’s the off target effects. In other words, changes to genes you didn’t expect. You don’t want to fix one problem, only to cause something else. IIRC, the reason why the integration errors were such a big deal is because they made it to the germline of the animal and could be transferred to offspring. This would have been extensively validated given the amount of press and regulatory pressure.
- maxerickson 7y agoThat isn't what they are doing. They are extracting bone morrow and editing that and then reintroducing it. It's basically a bone marrow transplant using the patient's edited stem cells. http://www.crisprtx.com/programs/hemoglobinopathies http://www.crisprtx.com/programs/hemoglobinopathies Words like "credible" and "unexpected" sort of make it impossible to answer the question. The doctors probably expect some editing errors and things like that. They are also doing a really small study to see what happens.
- tyingq 7y agoWords like "credible" and "unexpected" sort of make it impossible to answer the question I guess I could reword. Is there more than a remote chance the patient dies because of an error related to the gene editing?
- maxerickson 7y agoThey don't know for certain, hence a small pilot study. They of course believe that it will work and that there is a limited chance of such things.
- dekhn 7y agoTo know that with any confidence, you'd have to do this for a lot of people and look at the stats. We don't currently have the technology to answer a question like that convincingly for n=1 studies.
- mostlyjason 7y agoBack in 2017, 60 minutes covered a group that was cured of sickle cell disease with gene therapy. How does this approach differ when they already showed success? https://www.cbsnews.com/news/could-gene-therapy-cure-sickle-cell-anemia-60-minutes/# https://www.cbsnews.com/news/could-gene-therapy-cure-sickle-...
- el_cujo 7y agoJust skimming through the article you posted, it says trials with a similar treatment resulted in one patient dying from a massive immune response and two other got cancer. Sounds like it relies on a virus to deliver the DNA, but I didn't read too carefully, so maybe CRISPR will turn out to have fewer side effects or a greater success rate. I'm pretty sure one of the up-sides to CRISPR is compared to other gene-editing techniques it's relatively cheap, which is always a plus.
- maxerickson 7y agoThe death and cancers mentioned over there were from gene therapy trials in general, not the specific study in the link.
- maxerickson 7y agoThis study uses a different vector and targets a different gene. The NIH study uses a modified HIV to introduce the complete gene for the defective protein. https://www.nhlbi.nih.gov/news/2019/sickle-cell-patients-recovery-after-gene-therapy-heightens-hopes-cure https://www.nhlbi.nih.gov/news/2019/sickle-cell-patients-rec... This study uses CRISPR/Cas9 to increase expression of a gene that the patients already have.
- dghughes 7y agoFrom what I recall CRISPR is always used with another technology allowing more precision. CRISPR is too broad so it needs something with more precision to assist. I skimmed the article and did a quick search but I can't recall the name of the second method.
- adventured 7y agoAre you thinking of eg CRISPR Cas9 and or Cpf1? https://en.wikipedia.org/wiki/CRISPR#Cas9 https://en.wikipedia.org/wiki/CRISPR#Cas9
- krick 7y agoReminds me: this guy used gene therapy to cure himself of lactose intolerance quite a while ago. Not sure how common it actually is among biohackers. https://www.youtube.com/watch?v=J3FcbFqSoQY https://www.youtube.com/watch?v=J3FcbFqSoQY
- dekhn 7y agoPeople who do this are usually irresponsible and don't even know whether they've achieved their goal or not. """Just to make sure this is really clear because people have been angry about this ever being tested in more humans. This will not be used in humans again until significantly more testing is done and the manufacturing protocol is massively refined. This could mean years before another test is conducted. More tests will need to be done in animal models as well. Maybe we'll do away with the viral aspect all together and completely overhaul the DNA. The reason this video was posted was because I want this to be an open source technology. If you have suggestions for how it can be improved, leave it in the comments.""" I spent 20 years of my life working towards gene therapy and to see idiots do stuff like this (in an incompenent and unconvincing way) is so frustrating.
- 0xffff2 7y agoYou didn't actually tell us why it's irresponsible. Is there a real risk of them hurting anyone but themselves?
- anhner 7y agoI would guess that people would try and replicate their results and end up hurting themselves, for one.
- whatshisface 7y agoKilling yourself harms more people than just yourself, true personal independence is an anarcho-capitalist fantasy. The harms to others include your friends and loved ones being upset that you're dead (obviously), whatever it is you're testing gaining a reputation for killing people, and also it removes your presumably smart brain from the team of people working on the problem. If you merely harm yourself then all of the above apply but to a lesser degree.
- OJFord 7y agoFor a second I was really pleased to see a 'decline and go to plain text site' button on the cookie mask, but it goes to a page with only links to 'top news stories' etc. and not the actual article. What's the point?
- indienkid 7y agoFYI a little misleading that it's posted now, since the linked article came out in July.
- rubicon33 7y agoDoes anyone know of any companies hiring software engineers in this field? It's basically my dream job to somehow combine my interest in DNA/Genes with my skillset (programming)
- 8iterations 7y agoWe make a variant caller, which is used in the pipeline for this kind of work, you might find it an interesting rabbit hole. https://magnolia.sh https://magnolia.sh
- deleted 7y ago[deleted]
- lildoggo 7y agoI used to intern for a company called FlowJo that does DNA flow cytometry data analysis, which helps power projects like these. They are hiring a few engineers right now -> https://jobs.bd.com/search-jobs/Ashland%2C%20OR/159/4/6252001-5744337-5733351-5711789/42x19458/-122x70948/50/2 https://jobs.bd.com/search-jobs/Ashland%2C%20OR/159/4/625200... Here's their homepage https://www.flowjo.com/about/company https://www.flowjo.com/about/company
- jfarlow 7y agoIn the bay area: We are! (Serotiny): https://serotiny.bio https://serotiny.bio Synthego: https://www.synthego.com/ https://www.synthego.com/ Mammoth: https://mammoth.bio/ https://mammoth.bio/ Zymergen: https://www.zymergen.com/ https://www.zymergen.com/ Boston: Arbor: https://arbor.bio/ https://arbor.bio/ Asimov (kind of): https://www.asimov.io/ https://www.asimov.io/ Ginkgo: https://www.ginkgobioworks.com/ https://www.ginkgobioworks.com/ Also just happy to chat about the (very interesting, vibrant, if still tiny) intersection of software & synbio.
- impatientduck 7y agoI can't wait for the first mad scientist who figures out that CRISPR can be used for genetic warfare. Oh you're a <Palestinian/Israeli>? Well you just get a bad cold. Oh, you're a <Israeli/Palestinian>? Now you have ebola and your children are sterile and have down syndrome. Isn't science fun?
- btfreeman 7y agoSarah Cannon, where this trial took place, purchased Boston based Genospace ~18 months back. Curious of Genospace's role in this and if they are able to add value to any other Sarah Cannon trials.
- longliveTrump 7y agothis is absolutely wrong gene belongs to god us should stop such research, full stop
- deleted 7y ago[deleted]
- pmarreck 7y agoShe is literally GMO. So can we stop it with the anti-GMO-at-all-costs worldview?
- SamBam 7y agoI'm generally pro GMO food, but that's not a sound argument at all.
- neolefty 7y agoEditing human DNA will intensify the debate compared to editing crop plant DNA, I am sure. It's a little easier in this case since it's not a heritable change (only bone marrow DNA was edited), but as others have pointed out elsewhere, heritable changes would be a much bigger deal.
- intsunny 7y agoThat article was from July 2019, there is an update from today: https://www.npr.org/sections/health-shots/2019/10/10/766765780/after-a-life-of-painful-sickle-cell-disease-a-patient-hopes-gene-editing-can-hel https://www.npr.org/sections/health-shots/2019/10/10/7667657... The patient gets to go home today.
- sunstone 7y agoIt would be interesting to see the legal release this patient had to sign to get this done in the US.