Gene Therapy Cures Different X-Linked Disease.

[monkeybus]

Fantastic.

Researchers successfully treat babies with 'bubble boy' disease using gene therapy

amp.usatoday.com/amp/350238...

How the therapy works

The gene therapy works like this: A deactivated virus is inserted into the patient’s bone marrow, which delivers the correct gene copy into blood stem cells, replacing the defective one. These cells are then frozen and undergo testing.

“This virus is able to effectively deliver a healthy copy of the gene into a stem cell in a way that was not possible before,” Mamcarz said.

The patient then receives two days of low-dose busulfan, a chemotherapy drug that makes space in the marrow for the stem cells to grow, and the cells are then infused back into the patient.

And the kids are all cured.

I remember this disease, John Travolta was in the film -

imdb.com/title/tt0074236/?r...

And, forget not, Crispr has already cured some genetic diseases, and there are some new drugs in the pipeline, like the Minorix ones.

Oh yes, pack this site up. We are as good as cured.

[jayxie]

The fundamental solution to genetic disorders is gene therapy, which involves the introduction of normally expressed genes into the virus to improve symptoms. Our country is also preparing to start ald gene therapy trials, but only in children. I want to know, if the children are successful, will the same approach help us as adults, will the nerves that we've already damaged get better as a result.

[monkeybus in reply to jayxie]

You never know. The human body has tremendous power to repair itself.

[jayxie in reply to monkeybus]

We look forward to the early maturation of gene therapy for the benefit of our rare patients

[tieaknot]

Such exciting developments going on! Every once in a while in medicine they hit on “gamechangers” (like when Penicillin arrived on scene as an antibiotic). I think the new immunotherapies for cancers and the gene therapies are the next level “gamechangers”! That said time will tell what side effects and consequences come as a result (there are always strings attached, aren’t there???)

But it’s so nice to have new hope!!!

[julie_]

Agree that this is all very exciting! I am also curious if this would work with adults already affected.