Huntington’s disease treated for first time using gene therapy

Elie Dolgin • September 25, 2025

Preliminary results from a small trial offer the clearest evidence yet that the brain disease’s progression can be slowed.

A one-time gene therapy can markedly slow the progression of Huntington’s disease, potentially paving the way for the first ever treatment to alter the course of this rare, inherited brain disorder.


Continue reading at Nature.

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