Charting the translational pathway: ISSCR best practices for the development of PSC-derived therapies
Role of innate immune and inflammatory signaling in West Nile virus tropism and neuronal and glial cell death
Basket trials in rare diseases: a systematic review of current practices, methodological challenges, and future directions
Perineural delivery of AAV2/9 in non-human primates is a safe and efficient route for gene therapy in Charcot-Marie-Tooth diseases
Generation and characterization of three human induced pluripotent stem cell lines from patients with glycogen storage disease type II
