Preliminary but promising gene therapy shows 75% reduction in Huntington's disease progression

ABC News ^ | 9-25-25 | Dr. Noor Shaik

[MarlonRando]

The new therapy works by using a harmless virus to deliver special genetic instructions to the brain, researchers said. These instructions help turn off the harmful protein that damages brain cells. Unlike current treatments that only manage symptoms, AMT-130 is designed to be a one-time treatment that lasts a lifetime.

"I am thrilled that this study showed statistically significant effects on disease progression," professor Sarah Tabrizi, lead scientific advisor for the trial at

[MarlonRando]

Basically, 6 injections are made into the affected brain. The neurons, which manufacture the harmful protein are now reprogrammed to also produce a protein which slows the manufacture of the injurious protein. So symptoms can be delayed for decades.

[zeestephen]

Good news, but...

This treatment also requires 12-16 hours of delicate brain surgery.

I am not clear if that is one procedure, or multiple procedures.

In any event, any time you operate on the human brain, there are significant collateral risks.

[Valpal1]

The gene therapy is injected directly into various parts of the brain, so yes, it has risks. Which are smaller than the 100% certainty of a slow crumbling death from Huntington’s.

[fella]

What do they mean by gene therapy?

The Covid gene therapy has not turned out to be so good.

[redinIllinois]

Thanks for posting!