Neuronal Ceroid Lipofuscinosis are a group of devastating neurodegenerative lysosomal storage disorders that begin in childhood. Mutations in CLN3 gene lead to a NCL called Batten disease, characterized by the progressive loss of vision, movement, and cognition.
Updated: Jim Wilson to step down from gene therapy post at UPenn, will form two new companies
Gene therapy pioneer Jim Wilson will leave his academic post at the University of Pennsylvania and launch two new companies, one focused on developing rare
August 2, 2024