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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Bibliographische Detailangaben
Hauptverfasser: Noam DIAMANT, Einat NISSIM-ELIRAZ, Liron ELKOUBY
Format: Patent
Sprache:eng ; heb
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Beschreibung
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.