Gene therapy trials treat inherited blindness and deafness

Scientists have restored vision and hearing in people born with
congenital blindness or deafness in two separate gene therapy trials

https://www.dw.com/en/gene-therapy-trials-treat-inherited-blindness-and-deafness/a-69006407

On Sat, 11 May 2024, JAB wrote:

> Gene therapy trials treat inherited blindness and deafness
>
>
> Scientists have restored vision and hearing in people born with
> congenital blindness or deafness in two separate gene therapy trials
>
> https://www.dw.com/en/gene-therapy-trials-treat-inherited-blindness-and-deafness/a-69006407
>

I wonder when we'll see gene therapy treatments to improve human
functioning above a healthy baseline, instead of just treating deceases?

Also, with gene therapy, don't you have to "refill" every couple of years?
Of course that's a massive improvement over taking medication daily, and
depending on the treatment, maybe you don't have to, but I have a vague
memory reading somewhere that the gene therapy results get "flushed out"
of the system when cells die and your "original" ones are use to replace
them.

Not a scientist so caveat emptor as always! ;)

On Sun, 12 May 2024 12:58:47 +0200, D <nospam@example.net> wrote:

>gene therapy treatments

It's called "gene therapy trials," not gene therapy treatment.

So, this is essential "let's see what happens" research.

Medicine has come a long way since 1950, but has not crossed the
finished line. There is still too much statistical "research"
happening.

Some people can do this without consequences, but others cannot.
Researchers can say statistically, this affects X percent, but they
can't nail down the specifics as to why.

On Sun, 12 May 2024, JAB wrote:

> On Sun, 12 May 2024 12:58:47 +0200, D <nospam@example.net> wrote:
>
>> gene therapy treatments
>
> It's called "gene therapy trials," not gene therapy treatment.
>
> So, this is essential "let's see what happens" research.
>
> Medicine has come a long way since 1950, but has not crossed the
> finished line. There is still too much statistical "research"
> happening.
>
> Some people can do this without consequences, but others cannot.
> Researchers can say statistically, this affects X percent, but they
> can't nail down the specifics as to why.
>

I think there is at least one gene therapy treatment in production. I read
about it at least half a year ago in the mainstream newspaper, but can't
remember the details. That was in sweden and it was some where niche
decease.

On Mon, 13 May 2024 11:54:22 +0200, D <nospam@example.net> wrote:

>gene therapy treatment

What is gene therapy used to treat?

Gene therapy aims to fix a faulty gene or replace it with a healthy
gene to try to cure disease or make the body better able to fight
disease. It holds promise as a treatment for a wide range of diseases,
such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia
and AIDS.

The U.S. Food and Drug Administration (FDA) has approved gene therapy
products for several conditions, including cancer, spinal muscular
atrophy, hemophilia and sickle cell disease. But for most people, gene
therapy is available only as part of a clinical trial.

https://www.mayoclinic.org/tests-procedures/gene-therapy/about/pac-20384619