abstract |
Provided herein are methods and compositions for treating Batten's disease. Such compositions include recombinant adeno-associated virus (rAAV), the rAAV containing the AAV capsid, and the vector genome packaged therein, which are (a) AAV 5'reversed ends (a) ITR) sequence; (b) promoter; (c) CLN2 coding sequence encoding human TPP1; (d) AAV 3'ITR. Also provided herein are methods of treating Batten's disease, comprising administering the rAAV described herein to a subject in need thereof by multiple routes. Also provided herein are pharmaceutical compositions comprising rAAV described herein and related methods of treating Batten's disease. [Selection diagram] FIG. 1A |