HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a human chromosome 9 open reading frame 72 (C9orf72) gene, as well as methods of inhibiting expression of a C9orf72 gene and methods of treating subjects having a C9orf72-associated disease or disor...

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Bibliographische Detailangaben
Hauptverfasser: DANG, Lan, NGUYEN, Tuyen, SHARMA-KANNING, Aarti, SAVAGE, Brittany, MCININCH, James, FRENDEWEY, David
Format: Patent
Sprache:eng
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