DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF RPE65

RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-49516 and compositions, methods, and uses thereof.

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Bibliographische Detailangaben
Hauptverfasser: GOLAN MASHIACH, Michal, IZHAR, Lior, GEORGESON, Joseph, BARAM, David, EMMANUEL, Rafi, HERMAN, Asael
Format: Patent
Sprache:eng ; fre ; ger
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Zusammenfassung:RNA molecules comprising a guide sequence portion having 17-25 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-49516 and compositions, methods, and uses thereof.