Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates
No treatment is available for early-onset forms of metachromatic leukodystrophy (MLD), a lysosomal storage disease
