Caroline Sevin

Adeno-associated virus as a delivery vector for gene therapy of human diseases

Durable and efficient gene silencing in vivo by hit-and-run epigenome editing

The Challenge of Gene Therapy for Neurological Diseases: Strategies and Tools to Achieve Efficient Delivery to the Central Nervous System

Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates