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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Main Authors DROGUETT, GUSTAVO, ZAMBROWICZ, BRIAN, MILSTEIN, STUART, FISHILEVICH, ELANE, SHARMA-KANNING, AARTI, DUBOSE, BRITTANY, ZIMMERMANN, TRACY, MCININCH, JAMES D, GAGLIARDI, ANTHONY, FRENDEWEY, DAVID, CHIAO, ERIC, BROWN, KIRK
Format Patent
LanguageChinese
English
Published 01.11.2021
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
DERIVATIVES THEREOF
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
NUCLEIC ACIDS
NUCLEOSIDES
NUCLEOTIDES
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
SPIRITS
SUGARS
VINEGAR
WINE
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Summary: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 disorder, e.g., C9orf72 amyotrophic lateral sclerosis/frontotemporal dementia or Huntington-Like Syndrome Due To C9orf72 Expansions, using such dsRNAi agents and compositions.
Bibliography:Application Number: TW20209143671