DEPARTMT OF HEALTH AND HUMAN SERVICES;THE UNITED STATES OF AMERICA; AS REPRESENTED BY THE SECRETARY; DEPARTMENT OF HEALTH AND HUMAN SERVICES
发明人:
CHIORINI, JOHN A.,DI PASQUALE, GIOVANNI,VENDITTI, CHARLES,CHANDLER, RANDY
申请号：
CA2985786
公开号：
CA2985786A1
申请日:
2016.05.12
申请国别(地区):
CA
年份:
2016
代理人:
摘要：
The invention provides an adeno-associated viral (AAV) vector comprising a capsid comprising the amino acid sequence of SEQ ID NO: 4 or SEQ ID NO: 9, wherein the AAV vector further comprises a heterologous nucleic acid sequence, and wherein the heterologous nucleic acid sequence can encode the NGF-PTH fusion polypeptide or methylmalonyl CoA mutase enzyme. The invention also provides a polypeptide comprising nerve growth factor (NGF) signal peptide and parathyroid hormone (PTH), wherein the polypeptide can comprise, consist essentially of, or consist of the amino acid sequences of SEQ ID NO: 1 and SEQ ID NO: 2. The invention provides a nucleic acid encoding the polypeptide, a vector comprising the nucleic acid, and a composition comprising the polypeptide, nucleic acid, or vector, as well as treatment methods comprising the polypeptide, nucleic acid, vector, or composition. The invention further provides a method of treating methylmalonic acidaemia (MMA) in a mammal comprising administering an AAV vector comprising a heterologous nucleic acid sequence encoding methylmalonyl CoA mutase enzyme to the mammal.
