Board of Regents of the University of Nebraska;Ohio State Innovation Foundation
发明人:
Kielian, Tammy,Foust, Kevin
申请号：
AU2015364636
公开号：
AU2015364636A1
申请日:
2015.12.16
申请国别(地区):
AU
年份:
2017
代理人:
摘要：
Compositions and methods for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL), also known as Juvenile Batten Disease, are provided herein. In certain embodiments the compositions include but are not limited to adeno-associated viral (AAV) constructs, including self-complementary adeno-associated viral (sc-AAV) constructs, that express the human gene CLN3 (or a CLN3 cDNA).
