Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

GBA Variants and Parkinson Disease: Mechanisms and Treatments

dCas9-Based Scn1a Gene Activation Restores Inhibitory Interneuron Excitability and Attenuates Seizures in Dravet Syndrome Mice

AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy