Patent ID: 9149543
Filing Date: 2015-10-06
Classification: A61K,A61P

Abstract:
1. A method for non-viral delivery of a nucleic acid into the neuronal and non-neuronal cells of the central nervous system (CNS) of a subject to protect neuronal and non-neuronal cells from ischemic or traumatic injury, wherein the nucleic acid encodes a mono- or poly-peptide when operably-linked to a promoter, the method comprising: complexing the nucleic acid encoding a mono- or polypeptide when operably-linked to a promoter with a cationic, lipid congener to form a lipid-based nucleic acid lipoplex or vector to protect and stabilize the nucleic acid; and intrathecally administering the lipoplex to the cerebrospinal fluid (CSF) of the subject to deliver the protected, stable and active nucleic acid to the neuronal and non-neuronal cells of the CNS of the subject, wherein the subject is a primate or a human; and wherein the copy number of the nucleic acid in the neuronal and non-neuronal cells of the CNS is at least 5-fold higher than in cells outside the CNS.