Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
AssigneeThe Medical College of Wisconsin, Inc.
InventorsJeffrey A. Medin, Daniel H. Fowler, Murtaza S. Nagree, Tania Felizardo
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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.
Claims9
A method of producing a population of transduced T-Rapa cells that express an enzyme encoded by a transgene of interest for the treatment of a lysosomal storage disorder, the method comprising:
- (a) conditioning T-cells from a subject or suitable donor with rapamycin ex vivo to generate T-Rapa cells;
- and
- (b) transducing the T-Rapa cells in vitro with a vector comprising the transgene of interest to produce a population of transduced T-Rapa cells.
Dependencies are inferred from the claim text.