OLIGONUCLEOTIDES AND COMPOSITIONS THEREOF FOR NEUROMUSCULAR DISORDERS

Disclosed herein are engineered DUX4-targeting oligonucleotides for selective inhibition of RNA transcripts associated with a neuromuscular disease such as facioscapulohumeral muscular dystrophy. Also disclosed are vectors containing any of these, pharmaceutical formulations containing any of the th...

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Bibliographic Details
Main Authors BELGARD, Grant, SALEH, Anthony, MARUSAK, Charles, PLACE, Robert, MUNZ, Marton
Format Patent
LanguageEnglish
Published 29.02.2024
Subjects
BEER
BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
INFORMATION AND COMMUNICATION TECHNOLOGY [ICT] SPECIALLY ADAPTEDFOR SPECIFIC APPLICATION FIELDS
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PHYSICS
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
VINEGAR
WINE
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Summary:Disclosed herein are engineered DUX4-targeting oligonucleotides for selective inhibition of RNA transcripts associated with a neuromuscular disease such as facioscapulohumeral muscular dystrophy. Also disclosed are vectors containing any of these, pharmaceutical formulations containing any of the these, and kits containing any of the these. Also disclosed herein are methods of selectively inhibiting polypeptide expression and activity by contacting a DUX4-targeting oligonucleotide with an RNA transcript associated with a neuromuscular disease such as facioscapulohumeral muscular dystrophy.
Bibliography:Application Number: AU20220312501