COMPOSITIONS, METHODS AND APPLICATIONS OF GENE TRANSFER FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES Russian patent published in 2023 - IPC A61K31/343 A61K31/5377 A61K31/7008 A61K35/76 A61K38/13 A61K9/00 A61P25/00 A61P43/00 

FIELD: gene therapy.

SUBSTANCE: method intended for the treatment of lysosomal storage disease (LSD). A method for treating a mammal with lysosomal storage disease (LSD) involves the introduction of multiple AAV particles into the rostral lateral ventricle or caudal lateral ventricle of a mammal. These AAV particles contain (i) AAV capsid protein; (ii) a nucleic acid put between a pair of AAV inverted terminal repeats (ITRs), this nucleic acid encodes a polypeptide having lysosomal hydrolase activity; and (iii) an expression control element that controls the expression of this nucleic acid. These AAV particles are capable of transducing cells of this mammal and allowing expression of polypeptide, wherein said polypeptide comprises tripeptidyl peptidase-1 (TPP1), a proenzyme, or an enzymatically active option thereof.

EFFECT: treating diseases associated with lysosomal storage disease.

56 cl, 6 dwg, 2 tbl, 9 ex