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US 12,239,693 B2Granted
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.
Claims12
A method comprising:
- (a) conditioning T-cells from a subject with rapamycin ex vivo to generate T-Rapa cells, wherein the subject is suffering from Fabry disease;
- (b) transducing the T-Rapa cells in vitro with a vector comprising a transgene of interest that encodes an enzyme associated with a lysosomal storage disorder, wherein the enzyme associated with the lysosomal storage disorder is a-galactosidase A (α-gal A);
- (c) expanding the vector-transduced T-Rapa cells by culturing in vitro;
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- (d) administering the expanded transduced T-Rapa cells of step (c) derived from the subject to the subject by intravenous infusion, wherein the T-Rapa cells express the enzyme associated with the lysosomal storage disorder in the subject and reduce a level of globotriaosylceramide (Gb3) in the subject.
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Dependencies are inferred from the claim text.