Invention Grant
- Patent Title: Compositions and methods for treatment of GCASE related disease states
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Application No.: US16860319Application Date: 2020-04-28
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Publication No.: US11260106B2Publication Date: 2022-03-01
- Inventor: Ying Sun
- Applicant: Children's Hospital Medical Center
- Applicant Address: US OH Cincinnati
- Assignee: Children's Hospital Medical Center
- Current Assignee: Children's Hospital Medical Center
- Current Assignee Address: US OH Cincinnati
- Agency: Frost Brown Todd LLC
- Agent Nicole M. Tepe
- Main IPC: A61P25/28
- IPC: A61P25/28 ; A61K38/47 ; A61K9/00 ; A61K38/17 ; A61K45/06 ; C12N7/00 ; A61K48/00

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.
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