Compositions and methods for treatment of GCase related disease states
AssigneeChildren's Hospital Medical Center
InventorYing Sun
Life of this patent
Estimating the term…
Abstract
Disclosed are compositions and methods of treating a neurodegenerative disease in an individual. The methods disclose administration of an Integrin α4β1, Very Late Antigen-4 positive neural precursor cell (“VLA4+NPC”) transfected with a lentivirus overexpressing wild type GCase to an individual having a neurodegenerative disorder. The neurodegenerative disease may include lipid storage diseases, for example Gaucher disease, Parkinson's disease (PD), Dementia with Lewy bodies.
Claims11
A method of treating Gaucher disease in an individual in need thereof, comprising administering to said individual an Integrin α4β1, Very Late Antigen-4 positive neural precursor cell (“VLA4+NPC”), said VLA4+NPC being transfected with a lentivirus comprising wild type glucocerebrosidase (GCase).
Dependencies are inferred from the claim text.