Gene Therapy Research Review Issue 6

In this issue:

OTOF gene therapy vs. cochlear implantation in congenital deafness
Gene therapy using an AAV9 vector in infantile-onset Pompe’s disease
AAV8-mediated gene therapy in glycogen storage disease type 1a
OpenCRISPR-1: an AI-generated gene editor
Oligodendrocyte-targeted AAV gene therapy in Canavan disease
Toxicities & dynamics of CAR T-cell therapy in SLE vs. B-NHL
Dual-vector rAAVrh8 gene therapy in Tay-Sachs & Sandhoff diseases
Immunodepletion to reduce anti-AAV9 antibody titres prior to gene therapy
Gene therapy in AIPL1-associated severe retinal dystrophy
13-year follow-up of AAV-mediated gene therapy in severe haemophilia B