公开(公告)号：US20220184188A1

公开(公告)日：2022-06-16

申请号：US17310378

申请日：2020-01-31

Applicant: SPARK THERAPEUTICS, INC.

Inventor： David William ANDERSON ,  Maryann TOTO ,  Sue E.I. DASEN

IPC: A61K38/48 ,  A61K48/00 ,  A61P37/06 ,  C12N15/86

Abstract: Disclosed herein are methods for treating a primate in need of tripeptidyl peptidase 1 (TPP1), comprising (a) providing a recombinant adeno-associated virus (AAV) vector comprising a nucleic acid encoding TPP1; and (b) administering an amount of the recombinant AAV vector to the central nervous system (CNS) of the primate, wherein the TPP1 is expressed in the primate.