AAMS Neurology & Neurosciences · Vol. 7 · Issue 3 · 2026-03-01
CRISPR-Based Gene Therapy for Huntington Disease: Phase I Safety Results
Braun M, Sato H, Volkov A
Charité University Medicine, Berlin, Germany; University of Tokyo Hospital, Tokyo, Japan; Institute of Neurology, Moscow, Russia
Abstract
First-in-human phase I trial evaluating the safety and pharmacodynamics of intrathecal CRISPR-based gene therapy targeting mutant huntingtin in 24 patients with early Huntington disease.
Keywords: CRISPR, Huntington disease, gene therapy, phase I, neurodegenerative
Full Text
First-in-human phase I trial evaluating the safety and pharmacodynamics of intrathecal CRISPR-based gene therapy targeting mutant huntingtin in 24 patients with early Huntington disease.
This article is published open access by AAMS Neurology & Neurosciences. The complete peer-reviewed text can be read on the journal page.
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