Viral targeting of hematopoietic stem cells
AssigneesMassachusetts Institute of Technology, The Regents of the University of California
InventorsMichael Birnbaum, Connor Dobson, Stephanie Gaglione, Kole Roybal, Cassandra Burnett
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Abstract
Disclosed herein are compositions of retroviruses and methods of using the same for gene delivery to a hematopoietic stem cell (HSC), wherein the retroviruses comprise a viral envelope protein comprising at least one mutation that diminishes its native function, a non-viral membrane-bound protein comprising a membrane-bound domain and an extracellular targeting domain.
Claims27
A method of delivering one or more nucleic acids to a hematopoietic stem cell (HSC), the method comprising:
- (i) providing a retrovirus comprising the one or more nucleic acids, a viral envelope protein comprising at least one mutation that diminishes its native function, and a non-viral membrane-bound protein comprising an extracellular targeting domain that binds to a protein on the surface of the HSC;
- and
- (ii) contacting the retrovirus with the HSC,
- thereby delivering the one or more nucleic acids to the HSC.
-23. (canceled)
A method of gene editing in a hematopoietic stem cell (HSC), the method comprising:
- (i) providing a retrovirus comprising one or more nucleic acids encoding a gene editing composition, a viral envelope protein comprising at least one mutation that diminishes its native function, and a non-viral membrane-bound protein comprising an extracellular targeting domain that binds to a protein on the surface of the HSC;
- and
- (ii) contacting the retrovirus with the HSC such that the one or more nucleic acids encoding a gene editing composition are delivered to the HSC,
- wherein the gene editing composition specifically targets a section of the chromosomal DNA of the HSC to cause a genetic modification.
-48. (canceled)
Dependencies are inferred from the claim text.