Ex vivo gene therapy for lysosomal storage disorders: future perspectives.
Edina PolettoAndrew Oliveira SilvaRicardo WeinlichPriscila Keiko Matsumoto MartinDavi Coe TorresRoberto GiuglianiGuilherme BaldoPublished in: Expert opinion on biological therapy (2023)
Clinical trials are already ongoing, and soon approved therapies for LSD based on ex vivo gene therapy approaches should reach the market.