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AssigneeThe United States of America, as represented by the Secretary, Department of Health and Human Services
InventorsNaoya Uchida, Juan J. Haro Mora, John F. Tisdale
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The invention is directed to a system comprising a lentivirus vector particle which encodes at least one guide RNA sequence that is complementary to a first DNA sequence in a host cell genome, a Cas9 protein, and optionally a donor nucleic acid molecule comprising a second DNA sequence. The invention also is directed to a method of altering a DNA sequence in a host cell using such a system, where the host cell can be in a human and the altered DNA can be of the human β-globin gene. The invention also is directed to a fusion protein comprising a Cas9 protein and a cyclophilin A (CypA) protein. The invention also is directed to sequences of vectors that can be used in the system and method.
A system comprising:
A vector having the sequence of any one of SEQ ID NOS:
A system comprising:
Dependencies are inferred from the claim text.