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Gene therapy restores hearing to children with inherited deafness

cosmosmagazine.com

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Re: Gene therapy restores hearing to children with inherited deafness

#161

Post by m3kw9 »

akoboldfrying wrote: post #39
dyauspitr wrote: post #12

Earlier quoted context omitted.

I can’t wait for a geneCAD where we for example, design wings for a human body and then it works out the rest and generates a CRISPR based AAV that you can inject yourself with. True race and sex changes too. Hopefully in the next 50 years but that’s wishful thinking.

Let's try to fix the world's vast number of actual, boring health problems before trying to add the equivalent of spoilers and chrome-plated hubcaps to perfectly healthy human bodies.

Na, this isn’t it, you one of them that talks let’s solve poverty before spending money on xyz

Re: Gene therapy restores hearing to children with inherited deafness

#162

Post by BurningFrog »

zac256 wrote: post #57
29athrowaway wrote: post #51

The genetic corrections are not inherited, right?

It's considered taboo/forbidden for genetic corrections to target the germline. I think that's an unfortunate misapplication of ethics, presumably due to fears about eugenics.

It's taboo in Western medicine.

I doubt the whole world will respect that.

Re: Gene therapy restores hearing to children with inherited deafness

#163

Post by groovy2shoes »

Almondsetat wrote: post #136
gurumeditations wrote: post #118

Earlier quoted context omitted.

Sex changes/gender transitions is an actual boring health problem. The inability to assume the correct gender for a trans person often leads to social rejection, violence, poverty, prostitution, murder, and suicide.

If through gene editing you could also erase someone's disphoria what option would you choose?

Erasing someone's dysphoria would also fundamentally change their identity and personality. If I were to suffer a head injury today that so altered my personality, the result would be very unpredictable. I could become a better person, or a worse one. I could lose my wife, my career, everything. But even if the outcome were positive, I wouldn't be me anymore. This me would cease to exist, replaced by a new one.

On the other hand, a bodily alteration is much more predictable, and, importantly, I'd still be me. I wouldn't become some other person.

This me has an instinct for self-preservation. Thus, if both options were available, I would absolutely choose transition over erasure.

Re: Gene therapy restores hearing to children with inherited deafness

#164

Post by wouldbecouldbe »

Almondsetat wrote: post #147
wouldbecouldbe wrote: post #139

Earlier quoted context omitted.

That’s like trying to cure depression by getting rich. It hardly ever works

What?

Cutting off body parts to treat body dysphoria is something that should be done with great care and rarely leads to solving the underlying mental problems.

Trans is a much discussed variant that is hard to have a normal talk about, but there are many more variants, for instance women hating their breast & wanting to remove them, I think most people here would agree that intensive psychological treatment are preferable to actually removing healthy body parts. But in the end an adult can do what they want.

Re: Gene therapy restores hearing to children with inherited deafness

#165

Post by ADeerAppeared »

BurningFrog wrote: post #154

Language nit: You can't "restore" hearing to those who never had it.

That's not just a nit.

The idea that these are people who are "broken" and need to be "fixed" is something a lot of Deaf people are very opposed to.

Re: Gene therapy restores hearing to children with inherited deafness

#166

Post by sooheon »

chris_t wrote: post #126
GOD_Over_Djinn wrote: post #56

Earlier quoted context omitted.

Discoveries like this should be shared with the world - not gatekept by rent-seeking venture capitalists. Wouldn’t you rather be Jonas Salk than Scrooge McDuck? https://en.wikipedia.org/wiki/Jonas_Salk > Salk was immediately hailed as a "miracle worker" when the vaccine's success was first made public in April 1955, and chose to not patent the vaccine or seek any profit from it in order to maximize its global distrib…

Rent-seeking has a technical definition; earning money from things you invented does not meet it. While Salk is obviously an inspiring example, a wise society should happily incentivize the development of useful technology.

Intellectual monopolies disincentivize development and incentivize rent seeking. Here, rent seeking is legal action and lobbying intended to protect and extend monopolies, instead of advancing science.

Other than economic logic, we have natural experiments to back this up: i.e. the steam engine patent granted to Watt and Boulton in 1772 and its chilling effect on engine duty improvement (and the explosion of progress after patent expiry), and the modern pharmaceutical industry developing most strongly precisely where chemical patents were not granted, in 19th C. continental Europe, and slowing down wherever patent protections were eventually introduced, at the request of rent seeking lobbyists.

https://fee.org/articles/do-patents-encourage-or-hinder-inno...

http://www.dklevine.com/papers/anew09.pdf

Re: Gene therapy restores hearing to children with inherited deafness

#167

Post by rguiscard »

m3kw9 wrote: post #158
gota wrote: post #2

This stuff seems miraculous. > "Through minimally invasive surgery, Shu injected adeno-associated virus (AAV) engineered to carry and deliver functioning copies of the human OTOF transgene, into the children’s inner ears. " Can't read that and not get curious, excited, for the future. Congratulations to all those involved in the research and thanks to all who contributed to it - including all taxpayers if public fund…

I thought you can’t just inject genes and then the body will start replicate it. Does it need periodic injections?

That is why the gene is put into a harmless virus which will injects the gene into cells. It takes advantage of what virus usually does.

Re: Gene therapy restores hearing to children with inherited deafness

#168

Post by _factor »

evilduck wrote: post #52
akoboldfrying wrote: post #47

Earlier quoted context omitted.

That's true if we assume unlimited resources and equal awareness/perceived social value of the two applications. But I think the former doesn't hold, and the latter is greatly influenced by sites like HN, where I'm disappointed to consistently (if not exclusively) see breathless proposals for ways to design shinier hubcaps whenever some promising new technology appears. Yes, they can and likely will both happen to so…

Cosmetic surgery for vanity has helped improve the techniques and procedures and even the number of skilled practitioners that can then help those with disfigurements and deformities.

And cigarettes have benefited the lung cancer research centers. Just because it works doesn’t make it the best course.

Re: Gene therapy restores hearing to children with inherited deafness

#169

Post by Almondsetat »

sillysaurusx wrote: post #159
Almondsetat wrote: post #136

Earlier quoted context omitted.

If through gene editing you could also erase someone's disphoria what option would you choose?

Isn’t it obviously good to get rid of someone’s problem? Otherwise it wouldn’t be a problem.

If your perception doesn't match your body, would you prefer to change the perception or change the body?

Re: Gene therapy restores hearing to children with inherited deafness

#170

Post by toufka »

m3kw9 wrote: post #158
gota wrote: post #2

This stuff seems miraculous. > "Through minimally invasive surgery, Shu injected adeno-associated virus (AAV) engineered to carry and deliver functioning copies of the human OTOF transgene, into the children’s inner ears. " Can't read that and not get curious, excited, for the future. Congratulations to all those involved in the research and thanks to all who contributed to it - including all taxpayers if public fund…

I thought you can’t just inject genes and then the body will start replicate it. Does it need periodic injections?

If the DNA is in the cell's nucleus, the DNA will be utilized to produce whatever gene is encoded in the DNA. But if the DNA is just floating around on its own it will not survive a cell division event and the material would need to be periodically reinjected to keep working. However, if the injected DNA is part of a full chromosome, it will be replicated when the cell divides - and will be permanent as long as the cell or its progeny survive.

Some viruses will just inject the DNA into cells, but will not become part of the cell's genome ("transient" transduction). Other viruses (like lentiviruses and these adeno-associated viruses [AAVs]) inject their DNA not just into the cells, but also have machinery that splices their payload DNA directly into the cell's chromosomes ("integrated"). The location in the genome of the splicing event is relatively random. Random is not necessarily great as it could interrupt other genes already in the chromosome. CRISPR is a now-famous tool that helps "integrate" DNA into a specific spot in the genome by being guided to a specific location with a small piece of a specific sequence.

Once the DNA is integrated, any cell, and any of the cell's progeny, will produce or "express" the gene on the delivered DNA. In this case, they delivered the 5991 characters of DNA associated with the OROF gene [1].

[1] https://en.wikipedia.org/wiki/Otoferlin

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