AAV2 PARTICLE COMPRISING AN AAV2 CAPSID PROTEIN AND A VECTOR COMPRISING A NUCLEIC ACID ENCODING A TRIPEPTIDYL PEPTIDASE 1 (TPP1) FOR USE IN TREATING LATE INFANTILE CEROID LIPOFUSCINOSIS (LINCL) IN A NON-RODENT MAMMAL BY INTRAVENTRICULAR INJECTION OR ICV DELIVERY.

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