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LENTIVIRAL PROTEIN DELIVERY SYSTEM FOR RNA-GUIDED GENOME EDITING
LENTIVIRAL PROTEIN DELIVERY SYSTEM FOR RNA-GUIDED GENOME EDITING
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机译:RNA指导的基因组编辑的慢病毒蛋白递送系统
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摘要
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.
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