Gene Therapy and ASOs Drive New Hope for Huntington’s Patients

a adăugat un sunet Health

2025-06-17 09:31:19

Huntington’s disease (HD) is a devastating inherited neurodegenerative disorder caused by a CAG trinucleotide repeat expansion in the HTT gene. This mutation leads to the production of a toxic form of huntingtin protein, causing progressive brain cell death—particularly in the striatum and cortex—resulting in motor dysfunction, psychiatric symptoms, and cognitive decline. With...

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