Schaffer, David V.,Ojala, David Stephen,Romero, Philip A.
申请号：
AU2018324477
公开号：
AU2018324477A1
申请日:
2018.08.22
申请国别(地区):
AU
年份:
2019
代理人:
摘要：
The present disclosure provides recombinant adeno-associated virus virions with variant capsid protein, where the recombinant AAV (rAAV) virions exhibit one or more of increased ability to cross neuronal cell barriers, increased infectivity of a neural stem cell, increased infectivity of a neuronal cell, and reduced susceptibility to antibody neutralization, compared to a control AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a neural stem cell or a neuronal cell in an individual. The present disclosure also provides methods of modifying a target nucleic acid present in a neural stem cell or neuronal cell.
