Marco A. PASSINI,Lamya S. SHIHABUDDIN,Catherine R. O'RIORDAN,Seng H. CHENG,Catherine R. ORIORDAN
申请号：
US14888385
公开号：
US20160074474A1
申请日:
2013.05.01
申请国别(地区):
US
年份:
2016
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 the spinal column or cisterna magna in a human subject； for example, a pediatric human subject. Viral particles comprising AAV9 capsids are contemplated.
