Passini, Marco A.,Shihabuddin, Lamya S.,ORiordan, Catherine R.,Cheng, Seng H.
申请号：
AU2013388083
公开号：
AU2013388083A1
申请日:
2013.05.01
申请国别(地区):
AU
年份:
2015
代理人:
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.
