Claire Clelland, MD, PhD
University of California San Francisco
CRISPR gene therapy for C9orf72 FTD/ALS
A mutant repeat expansion in the C9orf72 gene is the most frequent known genetic cause of FTD and ALS. Targeting the mutant C9orf72 gene may be the most powerful therapeutic intervention. Here we propose a set of innovative, novel experiments to address whether CRISPR gene editing can reverse pathology or only prevent it (Aim 1) and identify the most efficient particle class to deliver to post-mitotic human neurons (Aim 2), with the aim to accelerate CRISPR gene therapy for ALS to the clinic.