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Three people with inherited diseases successfully treated with CRISPR

newscientist.com

21–30 of 74 posts

Re: Three people with inherited diseases successfully treated with CRISPR

#21

This is such mind-blowing success, it makes me contemplate switching fields. Is it reasonably feasible to switch from IT to Biotech/Medical Research in the mid thirties? I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.

Anyone knows how to get into the field?

Re: Three people with inherited diseases successfully treated with CRISPR

#22
Smalll thing, Beta thalassaemia isn’t a disease — as far as I know. It’s a disorder or a syndrome.

Definition of disease: Resulting from a pathophysiological response to external or internal factors.

Definition of disorder: A disruption of the disease to the normal or regular functions in the body or a part of the body.

Edit: I have it myself (and my kids do too), and I often correct people on this. Since I rather have a disorder than a disease:)

Re: Three people with inherited diseases successfully treated with CRISPR

#24

But will the drug approval agencies allow genetically engineered therapies for anything disruptive? For example, they've been blocking the caries vaccine for decades.

They will once scientists start moving to china to conduct their research

Re: Three people with inherited diseases successfully treated with CRISPR

#25

This is such mind-blowing success, it makes me contemplate switching fields. Is it reasonably feasible to switch from IT to Biotech/Medical Research in the mid thirties? I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.

Anecdotal, but my cousin did exactly this. I will note, he is probably the most exceptional person I known of, beyond those classic geniuses everyone knows.

Interesting, can you give a bit more details what exactly his path was?

Re: Three people with inherited diseases successfully treated with CRISPR

#26

It's interesting that they did not just undo the inherited disease. I assumed that, especially with Sickle Cell where we have a good understanding of how it works, they would go into Chromosome 11 and put it back how it "should" be with CRISPR. But instead they apply a workaround, ensuring continued fetal haemoglobin production. The article does not mention whether that's because putting Chromosome 11 back with CRISP…

It is much easier to wreck things with Crisper (in this case the regulatory region that turns of fetal hemoglobin) that to really go in and alter one or a couple of specific base pairs. What they did is nice but the latter would be the holy grail.

Re: Three people with inherited diseases successfully treated with CRISPR

#27
post #21

This is such mind-blowing success, it makes me contemplate switching fields. Is it reasonably feasible to switch from IT to Biotech/Medical Research in the mid thirties? I had a friend with Fukutin-related limb-girdle muscular dystrophy R13 and am wondering if CRISPR could be the solution for her.

Anyone knows how to get into the field?

As an other commenter wrote, if you know some software engineering, you are already valuable to all biotech companies, so it's probably better to go there as an engineer first and pick up the biology on the side than to go back to school and waste 5 years of your life without providing real value.

Re: Three people with inherited diseases successfully treated with CRISPR

#28

Earlier quoted context omitted.

Anecdotal, but my cousin did exactly this. I will note, he is probably the most exceptional person I known of, beyond those classic geniuses everyone knows.

Interesting, can you give a bit more details what exactly his path was?

comp sci in prestigious european uni. Moved to us in early 2000s. Big nationwide datacenter stuff. Decided to become doctor. Did doctory stuff and floated around medical for a while before switching to psyc. Currently forensic psych for infamous US prison. Found that rather than patient hop in regular psyc practice, or drug up people in asylums, with trial cases spanning years you can really deep dive a person and do good work.

Re: Three people with inherited diseases successfully treated with CRISPR

#29
post #21

Earlier quoted context omitted.

Anyone knows how to get into the field?

As an other commenter wrote, if you know some software engineering, you are already valuable to all biotech companies, so it's probably better to go there as an engineer first and pick up the biology on the side than to go back to school and waste 5 years of your life without providing real value.

Bioinformatics is probably a good option too.

Re: Three people with inherited diseases successfully treated with CRISPR

#30

But will the drug approval agencies allow genetically engineered therapies for anything disruptive? For example, they've been blocking the caries vaccine for decades.

yes. here’s an extreme example, but i’d say that every monoclonal antibody therapy on the market today (11 of the top 20 biggest drugs this year) qualify.

https://www.nytimes.com/2019/10/09/health/mila-makovec-drug....

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