РИДЖЕНТС ОФ ЗЭ ЮНИВЕРСИТИ ОФ МИННЕСОТА (US);РИДЖЕНКСБИО ИНК. (US)
发明人:
МАКАЙВОР Р. Скотт (US),БЕЛУР Лалита Р. (US),ЛОУ Уолтер (US),ФЭЙРБЭНКС Кэролин (US),КОЗАРСКИ Карен (US)
申请号:
RU2015152546
公开号:
RU2015152546A
申请日:
2014.05.15
申请国别(地区):
RU
年份:
2017
代理人:
摘要:
A method to prevent, inhibit or treat one or more symptoms associated with a disease of the central nervous system by intrathecally, intracerebroventricularly or endovascularly administering a rAAV encoding a gene product associated with the disease, e.g., a mammal in which the gene product is absent or present at a reduced level relative to a mammal without the disease.