USE OF LENTIVECTOR-TRANSDUCED T-RAPA CELLS FOR AMELIORATION OF LYSOSOMAL STORAGE DISORDERS

Abstract: the present disclosure provides methods of treating lysosomal storage disorders, e.g., fabry disease, gaucher disease, farber disease, and pompe disease. the method comprises producing vector-transduced t-rapa cells that express a transgene of interest and administering the cells to a patient in need thereof. the t-rapa cells may be transduced with a dual promoter lentivirus vector.

Inventor(s): Jeffrey A. Medin, Daniel H. Fowler, Murtaza S. Nagree, Tania Felizardo

CPC Classification: A61K38/443 (PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms ; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles ; soap compositions ))

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