REGENXBIO INC.;REGENTS OF THE UNIVERSITY OF MINNESOTA
发明人:
MCIVOR, R. SCOTT,BELUR, LALITHA R.,LOW, WALTER,FAIRBANKS, CAROLYN,KOZARSKY, KAREN
申请号:
CA2912678
公开号:
CA2912678A1
申请日:
2014.05.15
申请国别(地区):
CA
年份:
2014
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.