Compositions and methods for treating spinal muscular atrophy

摘要

The present provides methods for treating spinal muscular atrophy using a self- complementary recombinant adeno-associated virus (rAAV) viral particle comprising a transgene expressing SMN. In one aspect, the viral particles are administered in the spinal column or cisterna magna in a human subject; for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.