Three people with inherited diseases successfully treated with CRISPR
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Re: Three people with inherited diseases successfully treated with CRISPR
#2This 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.
Re: Three people with inherited diseases successfully treated with CRISPR
#3It'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 CRISPR is harder, or whether for some reason that wouldn't fix the problem.
Re: Three people with inherited diseases successfully treated with CRISPR
#4It'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…
That's also what struck me as odd, but having maintained legacy systems for a long time I can see why it's sometimes preferable to use a proven if inelegant workaround than going in and try to fix the actual defect, especially if you have limited debugging options.
Re: Three people with inherited diseases successfully treated with CRISPR
#5This 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.
Go for it. We'll probably still be looking for a vaccine for corona by the time you graduate!
Re: Three people with inherited diseases successfully treated with CRISPR
#6This 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.
You don't even need to switch fields. There are plenty of biomedical companies (and what isn't a tech company these days?) and projects that could use some passionate technical people.
Re: Three people with inherited diseases successfully treated with CRISPR
#7It'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…
One reason may be that a general fix that produces good hemoglobin is much more efficient than attempting to fix the original problem. People can have a near-limitless number of mutations that lead to defective proteins, so creating fixes for all those "bugs" can be very expensive. A general fix that works around the "bug" is cheaper, consistant, and more scalable.
Another way to look at it: Would a per-patient fix be possible? Maybe. Is it worth debugging and fixing everyone's crappy DNA to do that? Probably not.
Re: Three people with inherited diseases successfully treated with CRISPR
#8But will the drug approval agencies allow genetically engineered therapies for anything disruptive? For example, they've been blocking the caries vaccine for decades.
Re: Three people with inherited diseases successfully treated with CRISPR
#9But will the drug approval agencies allow genetically engineered therapies for anything disruptive? For example, they've been blocking the caries vaccine for decades.
> blocking the caries vaccine for decades
Please give more details.
Re: Three people with inherited diseases successfully treated with CRISPR
#10This 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.
You don't even need to switch fields. There are plenty of biomedical companies (and what isn't a tech company these days?) and projects that could use some passionate technical people.
What are the biomedical FAANGs?