Lentiviral vectors for in vivo targeting of hematopoietic cells
The present application, in some embodiments, is directed to a synthetic polynucleotide including a nucleic acid sequence encoding a mutated vesicular stomatitis virus G (VSV-G) protein, being characterized by having reduced or no binding affinity to a low-density lipoprotein (LDL) receptor, compare...
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Zusammenfassung: | The present application, in some embodiments, is directed to a synthetic polynucleotide including a nucleic acid sequence encoding a mutated vesicular stomatitis virus G (VSV-G) protein, being characterized by having reduced or no binding affinity to a low-density lipoprotein (LDL) receptor, compared to a control VSV-G. Further provided is a viral vector including the mutated VSV-G protein, and optionally further includes a single-chain variable fragment (scFv). Further provided are, inter alia, compositions including the expression vector, and use thereof, such as for in vivo gene therapy in a subject in need thereof. |
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