发明名称 |
Gene therapy for neurometabolic disorders |
摘要 |
The disclosure pertains to methods for treating neurometabolic disorder including lysosomal storage diseases that affect the central nervous system, e.g., Niemann-Pick A disease. The disclosed methods involve contacting an axonal ending of a neuron with a composition containing high titer AAV carrying a therapeutic transgene so that the AAV vector is axonally transported in a retrograde fashion and transgene product is expressed distally to the administration site.
|
申请公布号 |
US2006171926(A1) |
申请公布日期 |
2006.08.03 |
申请号 |
US20050263664 |
申请日期 |
2005.10.31 |
申请人 |
PASSINI MARCO A;STEWART GREGORY R;DODGE JAMES |
发明人 |
PASSINI MARCO A.;STEWART GREGORY R.;DODGE JAMES |
分类号 |
A61K48/00 |
主分类号 |
A61K48/00 |
代理机构 |
|
代理人 |
|
主权项 |
|
地址 |
|