AAV VECTORS FOR GENE EDITING

Provided herein polynucleotides configured for incorporation into recombinant adeno-associated virus (AAV) vectors. The polynucleotides encode for CRISPR proteins, gRNA, and ancillary components of AAV vectors useful in the modification of target nucleic acids. The systems are also useful for introd...

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Hauptverfasser: STAAHL, Brett T, FORTUNY, Cecile, BANEY, Katherine, WRIGHT, Addison, DENNY, Sarah, SIDORE, Angus, MOHR, Manuel, ADIL, Maroof, MAKHIJA, Suraj, HIGGINS, Sean, OAKES, Benjamin
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung:Provided herein polynucleotides configured for incorporation into recombinant adeno-associated virus (AAV) vectors. The polynucleotides encode for CRISPR proteins, gRNA, and ancillary components of AAV vectors useful in the modification of target nucleic acids. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the target nucleic acid of a gene. Also provided are methods of using such AAV vectors to modify cells having such mutations.