Patent ID: 9579362
Date: 2017-02-28
CPC Classifications: A61K,A61P,C07K,C12N

Claim:
1. A method of treating Parkinson's disease in a human subject, said method comprising: administering by injection into the striatum of the brain of the subject suffering from Parkinson's disease a therapeutically effective amount of a polynucleotide encoding pre-(β)pro-GDNF protein, wherein the polynucleotide encoding the pre-(β)pro-GDNF protein is a polynucleotide encoding a polypeptide comprising at least one of the amino acid sequences selected from the group consisting of SEQ ID NO: 31, SEQ ID NO: 35, and SEQ ID NO: 52, wherein the polynucleotide is in a recombinant adeno-associated virus suitable to introduce the polynucleotide into brain cells, and wherein the polynucleotide is operatively linked to a cytomegalovirus promoter expression regulatory element, wherein the encoded pre-(β)pro-GDNF protein promotes the survival of dopaminergic neurons in Parkinson's Disease.