Building CRISPR Gene Therapies for the Central Nervous System

This narrative review discusses several types of gene editing using clustered regularly interspaced short palindromic repeats (CRISPR), how these are applied to different genetic variants, challenges of delivering genome editors to the central nervous system, ways to minimize the potential in vivo genotoxic effects of genome editors, the ethical considerations of germline editing, and the regulatory challenges of human-specific therapy.
Source: JAMA Neurology - Category: Neurology Source Type: research