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Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

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Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#11
post #2

I work in gene therapy; expect a lot of headlines like this in the coming years. We're just getting started with solving the easy-to-fix problems and disorders right now, but they're falling quickly. Soon it'll be possible to seriously talk about improving working functionality via gene therapy-- I'd expect simple musculoskeletal augmentation first.

Give us so more, you tease!

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#12
post #2

I work in gene therapy; expect a lot of headlines like this in the coming years. We're just getting started with solving the easy-to-fix problems and disorders right now, but they're falling quickly. Soon it'll be possible to seriously talk about improving working functionality via gene therapy-- I'd expect simple musculoskeletal augmentation first.

Cool, I expect it is the beginning of the end of genetic disease. What that means overall I'm not sure I can say but for people whose life can be improved dramatically by fixing a genetic mixup it will be literally life altering.

My sister is especially interested in gene therapy research associated with Downs Syndrome.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#13
post #8
post #6

Earlier quoted context omitted.

Could you describe a bit more about your job? I am currently in training. Any advice would he helpful. I am thinking of doing an PhD. Edit: Let me rephrase that: can I PM you somehow for more details and questions?

I didn't go the academic science route, but I can maybe tell you a bit about some people who did/are currently. I'll start at the very end, then work backwards: The professors: These guys lived the dream from start to finish. They now have multiple postdocs working to answer the scientific questions that interest them. The professors are responsible for fundraising and presenting the research, and are almost never in…

Good luck man, i wonder what the industry is doing differently than academia to speed up the process. Also what is the course of the most passionate people, academia or otherwise?

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#15

Does the strengthening of neural connections only work with people with that specific gene? Could this be extended to other cortical visual disorders, such as amblyopia?

As someone with glaucoma and bilateral amblyopia (I had congenital cataracts in both eyes) the answer to this interests me greatly!

I'd also add:

Will this kind of gene therapy allow protection or regeneration of the optic nerve?

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#16
post #2

I work in gene therapy; expect a lot of headlines like this in the coming years. We're just getting started with solving the easy-to-fix problems and disorders right now, but they're falling quickly. Soon it'll be possible to seriously talk about improving working functionality via gene therapy-- I'd expect simple musculoskeletal augmentation first.

Technically, we were just getting started with solving the easy to fix problems with gene therapy over twenty years ago.

The field was moribund- for a number of reasons- for quite some time. New results are great, but nothing truly transformative, and basically as hard as delivering classic pharma or biotech treatments.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#17
post #2

I work in gene therapy; expect a lot of headlines like this in the coming years. We're just getting started with solving the easy-to-fix problems and disorders right now, but they're falling quickly. Soon it'll be possible to seriously talk about improving working functionality via gene therapy-- I'd expect simple musculoskeletal augmentation first.

Any contact to get gene therapy for muscular dystrophy? :-p

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#18
post #2

I work in gene therapy; expect a lot of headlines like this in the coming years. We're just getting started with solving the easy-to-fix problems and disorders right now, but they're falling quickly. Soon it'll be possible to seriously talk about improving working functionality via gene therapy-- I'd expect simple musculoskeletal augmentation first.

Cool, I expect it is the beginning of the end of genetic disease. What that means overall I'm not sure I can say but for people whose life can be improved dramatically by fixing a genetic mixup it will be literally life altering. My sister is especially interested in gene therapy research associated with Downs Syndrome.

I can see gene therapy making significant progress in the next decade on diseases for which gene therapy can be effective, but there are some serious problems. you still have to get the therapy to the cells that need it; the delivery problem is hard. Further, it's not certain that you can easily identify which "patch" (to the extent the idea of patching the genome is actually a valid one) is going to work in an individual.

I woudl say that we will see the end of some genetic diseases, similar ot the way that vaccines reduced the incidence of some diseases. Other diseases, especially complex, multifactorial ones, we simply don't have the theory to understand their mechanism, and in general, you need to have some amount of mechanistic understanding to come up with therapies.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#19
post #8
post #6

Earlier quoted context omitted.

Could you describe a bit more about your job? I am currently in training. Any advice would he helpful. I am thinking of doing an PhD. Edit: Let me rephrase that: can I PM you somehow for more details and questions?

I didn't go the academic science route, but I can maybe tell you a bit about some people who did/are currently. I'll start at the very end, then work backwards: The professors: These guys lived the dream from start to finish. They now have multiple postdocs working to answer the scientific questions that interest them. The professors are responsible for fundraising and presenting the research, and are almost never in…

You have a pretty rosy view of professors. I don't think being a professor means you've won any academic games.

You don't get to take sabbaticals when you want. Professors have a lot of people to answer to- granting agencies, deans, students, and more.

Re: Gene Therapy Makes Near-Blind Patients See by Strengthening Neural Connections

#20
post #19
post #8

Earlier quoted context omitted.

I didn't go the academic science route, but I can maybe tell you a bit about some people who did/are currently. I'll start at the very end, then work backwards: The professors: These guys lived the dream from start to finish. They now have multiple postdocs working to answer the scientific questions that interest them. The professors are responsible for fundraising and presenting the research, and are almost never in…

You have a pretty rosy view of professors. I don't think being a professor means you've won any academic games. You don't get to take sabbaticals when you want. Professors have a lot of people to answer to- granting agencies, deans, students, and more.

>>Professors have a lot of people to answer to

Nope. The agencies you mentioned effect their ability to grow, their formal employer is the University. If their research goes to hell they can teach a class and make the same amount of money. Sometimes their students are completely free (TAs).

Having seen professors give projects they know dont work and falsify work under vagious euphemisms, they are the problem with academia - they provide little value but somehow run the show. This is especially true for less mathematically difficult fields like biology.

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