Gene therapy of storage disorders by retroviral and lentiviral vectors.
Biffi
Alessandra
A
San Raffaele Telethon Institute for Gene Therapy and Vita Salute University, H. San Raffaele Scientific Institute, Milan 20132, Italy.
Naldini
Luigi
L
D000818:Animals; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000235:genetics / Q000627:therapeutic use*; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D016464:Lysosomal Storage Diseases / Q000235:genetics / Q000628:therapy*; D009474:Neurons / Q000637:transplantation; D014161:Transduction, Genetic