COMPOSITIONS AND METHODS FOR TREATING CAG REPEAT DISEASES

The present disclosure provides a double-stranded RNA (dsRNA) comprising: a) a first strand that hybridizes to a target CAG repeat region of a CAG repeat containing RNA; and b) a second strand that hybridizes to the first strand, wherein the first strand comprises: i) a first mismatch to the target...

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Bibliographische Detailangaben
Hauptverfasser: JENSIK, Philip J, KOTTERMAN, Melissa A, BLEWETT, Megan, DE KLERK, Eleonora
Format: Patent
Sprache:eng ; fre
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