CRISPR-Based Gene Therapy for Huntington Disease: Phase I/II Trial Results

Abstract

We report first-in-human results of AAV-delivered CRISPR-Cas9 gene therapy targeting the mutant HTT gene in 18 Huntington disease patients. At 12 months, 40-65% reduction in mutant huntingtin protein levels was achieved.

Keywords: CRISPR, gene therapy, Huntington disease, clinical trial