Gene therapy for lysosomal storage disorders

Abstract

Gene therapy has become the next frontier in the treatment and potential cure of many disorders that are refractive to current therapies. The lysosomal storage disorders (LSDs) collectively constitute one of the largest groups of inherited metabolic disorders. Propelled by the exciting success of enzyme replacement therapies applied to LSDs without neuropathology, the development of effective gene therapy protocols for the LSDs is underway. For the LSDs with neuropathology, in particular, it has become clear that gene therapy is at present one of only a few therapeutic options with the potential for success. Studies summarised in this review indicate that gene therapy using a variety of vectors both in vivo and ex vivo have shown great promise for the treatment of these diseases. However, several problems require serious attention before it will be feasible to embark on human gene therapy trials.

• AAV
• adenovirus
• CNS
• enzyme replacement
• lentivirus