In a First, US Doctors Use Crispr Tool to Treat Patient with Genetic Disorder

https://www.npr.org/sections/health-shots/2019/07/29/744826505/sickle-cell-patient-reveals-why-she-is-volunteering-for-landmark-gene-editing-st

by mhb • 7 years ago

270 82 7 years ago

npr.org

tyingq2• 7 years ago

Assuming they use CRISPR outside the body, on blood, then infuse the blood back in...

Are there any credible risks that something unexpected happens?

AndrewDucker• 7 years ago

Yup: "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...

justinjlynn• 7 years ago

What'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.

maxerickson5• 7 years ago

That 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

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.

tyingq2• 7 years ago

Words 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?

mostlyjason• 7 years ago

Back 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-...

el_cujo• 7 years ago

Just 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.

maxerickson5• 7 years ago

The death and cancers mentioned over there were from gene therapy trials in general, not the specific study in the link.

maxerickson5• 7 years ago

This 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-rec...

This study uses CRISPR/Cas9 to increase expression of a gene that the patients already have.

dghughes• 7 years ago

From 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• 7 years ago

Are you thinking of eg CRISPR Cas9 and or Cpf1?

https://en.wikipedia.org/wiki/CRISPR#Cas9

krick5• 7 years ago

Reminds 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

dekhn5• 7 years ago

People 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.

0xffff22• 7 years ago

You didn't actually tell us why it's irresponsible. Is there a real risk of them hurting anyone but themselves?

krick5• 7 years ago

Well, he can eat pizza now. What use your 20 years of experience were to him anyways?

buboard• 7 years ago

interesting .. didn't know that it was already possible

Japhy_Ryder2• 7 years ago

He could've also just, y'know, stopped consuming garbage lactose.

samfriedman• 7 years ago

How can one tell the difference between good and "garbage" lactose, then?

dooglius• 7 years ago

There are some pretty big methodological issues here, and some of the comments make good cases that this didn't actually do anything.

pmarreck4• 7 years ago

cure himself of lactose intolerance

Which is funny, because lactose intolerance is actually the norm, before a bunch of ancient Europeans decided that they'd figure out how to live off cows and there was selection pressure to extend lactose tolerance out of infancy where it normally remains

SamBam2• 7 years ago

You keep using this word "normally"...

Are you saying that natural selection is not normal if it was only a few thousand years ago?

OJFord• 7 years ago

For 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• 7 years ago

FYI a little misleading that it's posted now, since the linked article came out in July.

rubicon333• 7 years ago

Does 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• 7 years ago

We 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

lildoggo• 7 years ago

I 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/159/4/625200...

Here's their homepage https://www.flowjo.com/about/company

jfarlow• 7 years ago

In the bay area:

We are! (Serotiny): https://serotiny.bio

Synthego: https://www.synthego.com/

Mammoth: https://mammoth.bio/

Zymergen: https://www.zymergen.com/

Boston:

Arbor: https://arbor.bio/

Asimov (kind of): https://www.asimov.io/

Ginkgo: https://www.ginkgobioworks.com/

Also just happy to chat about the (very interesting, vibrant, if still tiny) intersection of software & synbio.

btfreeman• 7 years ago

Sarah 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.

pmarreck4• 7 years ago

She is literally GMO.

So can we stop it with the anti-GMO-at-all-costs worldview?

SamBam2• 7 years ago

I'm generally pro GMO food, but that's not a sound argument at all.

neolefty• 7 years ago

Editing 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• 7 years ago

That article was from July 2019, there is an update from today:

https://www.npr.org/sections/health-shots/2019/10/10/7667657...

The patient gets to go home today.

sunstone• 7 years ago

It would be interesting to see the legal release this patient had to sign to get this done in the US.

In a First, US Doctors Use Crispr Tool to Treat Patient with Genetic Disorder

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