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Deaf girl is cured in world first gene therapy trial

independent.co.uk

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Re: Deaf girl is cured in world first gene therapy trial

#291

Post by surfingdino »

dekhn wrote: post #222
jasonfarnon wrote: post #221

Earlier quoted context omitted.

So you're only pessimistic about the deployment of germline modifications rather than therapies like this?

You missed the part where I'm describing germline modifications for non medical uses . Like, intelligence augmentation for your children. Very controversial.

Public acceptance of those modifications could increase if such treatments are marketed as new or alternative approaches to what the cosmetic surgery and erectile disfunction treatment industries are making money off today.

Re: Deaf girl is cured in world first gene therapy trial

#292

Post by kosmozaut »

contingencies wrote: post #198
alsetmusic wrote: post #71

Medical science is about the only thing in modern life that consistently delivers hope. While all the other terrible things are going on, something like this comes along and I’m just extremely happy to be alive right now.

Serious suggestion: try gardening. To plant a garden is to believe in tomorrow. - Audrey Hepburn

Thanks for this beautiful quote! So simple yet powerful

Re: Deaf girl is cured in world first gene therapy trial

#293

Post by spondylosaurus »

silverquiet wrote: post #220
spondylosaurus wrote: post #197

Earlier quoted context omitted.

Hip problems are brutal, been there before :( Mine (mostly) resolved when I addressed some other underlying issues but I was also seriously considering joint replacement for a bit! The surgery and downtime are no joke, but everyone I've talked to who went through with a replacement was glad they did. I even know at least one guy who now works on his feet all day. Not saying to just take the plunge now, but if you eve…

Yes - it’s a bit ironic that I have an extremely rare condition (Perthes disease) that ultimately may be resolved by a very common surgery. And it is known as one of the best as far as outcomes - the Lancet called it the surgery of the century, so I do at least have some hope there. Good to hear you were able to mostly resolve your issue. Given your name I’m assuming you have ankylosing spondylitis - I certainly see…

Damn, I recently learned about Perthes and it does not sound fun. Glad you're managing for now but I hope you get some relief in the future, through surgery or otherwise.

And yep, spondylitis with a side of hip bursitis, so Perthes hits close to home. Eventually I figured out I have colitis, started to treat that, and the arthritis got 90% better. Also I learned that some people with really aggressive colitis who go on steroids for years wind up with osteoporosis so bad that they need... hip replacement surgery, lol. It all comes full circle!

Re: Deaf girl is cured in world first gene therapy trial

#294

Post by pas »

hnick wrote: post #243
codeulike wrote: post #193

How it actually works (from https://www.fiercebiotech.com/biotech/little-protein-factory... ) DB-OTO is a cell-selective AAV gene therapy for children with hearing loss stemming from a mutation to the otoferlin gene. The otoferlin protein is expressed in the sensory hair cells of the ear, which have tiny cilia that move as vibrations come into the ear. These cells help signal between the auditory nerve and the hair c…

Sorry just wanted to clear this up - do you mean it delivers an extra set of DNA that does its thing alongside what is already there, rather than editing the main sequence like CRISPR wants to?

The usual term for this is "nonintegrating" meaning the virus (or whatever delivery mechanism) does not insert itself (or anything) into the cell's genome. (Some viruses do this to help them proliferate - and they do it crudely which increases cancer risk, - but some don't because it's added complexity to have this "feature".)

https://pubmed.ncbi.nlm.nih.gov/28895845/

Re: Deaf girl is cured in world first gene therapy trial

#295

Post by codeulike »

verisimi wrote: post #281
bglazer wrote: post #183

Earlier quoted context omitted.

It's a virus that adds a bit of genetic code to cells in the ear. The new genetic code is the instructions for the protein that is broken in the the little girl (and other similar patients). The cell can then use the newly introduced DNA to make a protein. This protein restores the link between the auditory sensory cells and the auditory nerve cells. Regarding the word "infused", the viral particles are carried in an…

Thanks for your answer. Yes, I get that bit you describe, more or less. But also cells die - all cells are new in the human body after 7 years, right? (Or are we saying ear cells don't?) New cells are produced. Whatever creates new ear cells, needs to know to produce them with the update. I know that marrow creates blood cells.... and I'm not sure how replacement ear cells are created, but if it were something like m…

These particular hair cells dont die, so they think the treatment will persist, see https://news.ycombinator.com/item?id=40313256

Re: Deaf girl is cured in world first gene therapy trial

#296

Post by codeulike »

dekhn wrote: post #130

Gene therapy has been an extremely slow game. Part of that is ethics, and part of that is technology. A single death in a single trial slowed down gene therapy for decades https://en.wikipedia.org/wiki/Jesse_Gelsinger I dedicated much of my biology training to learning how to carry out gene therapy only to conclude that it will continue to be the underdog in disease therapy for a long time. I do not expect to see lar…

I do not expect to see large-scale germline modification for non-disease purposes in my lifetime (another 20-30 years).

I had to read that twice. Non-disease purposes?

The term "Gene-therapy" to me means therapy as in treatment of a disease or condition. If we're talking about non-disease purposes like enhancements then you're damn right thats still a long way off. I would have put it further off than that, its going to be a minefield.

(Although with 200 different countries in the world it seems like someone will just do it anyway. Maybe someone is doing it now?)

Re: Deaf girl is cured in world first gene therapy trial

#297

Post by s_dev »

anthony__j wrote: post #14
screye wrote: post #7

Earlier quoted context omitted.

Science doesn't (and shouldn't) care for how people socialize around a disability. I recognize the power and a reclaimed self worth that comes with reframing narratives around deafness. The lack of hearing is a disease. Because diseases are nominatively about being at dis-ease. As long as it's difficult for deaf people to socialize with those who outside the community, it dis-eases them.

like someone mentioned in a separate comment, you are conflating disease with disability here. when people become too old to walk without assistance, you wouldn't say that they have a walking disease.

Yes but the arthritis or whatnot that causes the impairment is a disease.

Re: Deaf girl is cured in world first gene therapy trial

#298

Post by M95D »

harimau777 wrote: post #207
jajko wrote: post #166

Earlier quoted context omitted.

Astronomy is cool too, nothing anchors you back in reality like realizing how absolutely microscopic and insignificant our entire mankind's existence was and is. Solid perspective and understanding of bigger picture is important, then nothing can surprise you much.

Personally, I find astronomy quite depressing. Learning that humanity is stuck on this rock; at least for my lifetime if not forever. As a kid I dreamed of exploring space. As an adult I learned that is impossible.

I always knew I would see the first man on the moon. I never dreamed I would see the last. -- Jerry Eugene Pournelle

Re: Deaf girl is cured in world first gene therapy trial

#299

Post by wddkcs »

downWidOutaFite wrote: post #200
alsetmusic wrote: post #71

Medical science is about the only thing in modern life that consistently delivers hope. While all the other terrible things are going on, something like this comes along and I’m just extremely happy to be alive right now.

Funny because I feel the opposite. Health care is so broken with all the politics, insurance, bureaucracy, financialization, etc that the vast majority of people are not able to reap the rewards of progress. Even the science is perverted because of bad incentives and only potentially lucrative research is funded.

It couldn't exist otherwise. The privileged will always be the ones to push the boundaries of any endeavor, medicine included. Have hope that those rewards continue to work their way down, even though the process is long and arduous.

Re: Deaf girl is cured in world first gene therapy trial

#300

Post by Jensson »

codeulike wrote: post #296
dekhn wrote: post #130

Gene therapy has been an extremely slow game. Part of that is ethics, and part of that is technology. A single death in a single trial slowed down gene therapy for decades https://en.wikipedia.org/wiki/Jesse_Gelsinger I dedicated much of my biology training to learning how to carry out gene therapy only to conclude that it will continue to be the underdog in disease therapy for a long time. I do not expect to see lar…

I do not expect to see large-scale germline modification for non-disease purposes in my lifetime (another 20-30 years). I had to read that twice. Non-disease purposes? The term "Gene-therapy" to me means therapy as in treatment of a disease or condition. If we're talking about non-disease purposes like enhancements then you're damn right thats still a long way off. I would have put it further off than that, its going…

> If we're talking about non-disease purposes like enhancements

Like extending lifespan since death isn't seen as "disease", is probably what most people are after.

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