AAV2 particle comprising a AAV2 capsid protein and a vector comprising a nucleic acid encoding a tripeptidyl peptidase 1 (TPP1) for the treatment of late childhood ceroid lipofuscinosis (LINCL) in a non-rodent mammal by intraventricular injection or administration icv

摘要

The present disclosure provides methods of treating a disease in a non-rodent mammal comprising administering to the cerebrospinal fluid (CSF) of the non-rodent mammal an rAAV2 particle comprising an AAV2 capsid protein and a vector comprising a nucleic acid encoding a therapeutic protein inserted between a pair of AAV inverted terminal repeats in a manner effective to infect an ependymal cell in the non-rodent mammal, wherein the ependymal cell secretes the therapeutic protein so as to treat the disease