DUX4 INHIBITORS AND METHODS OF USE THEREOF

• Main Authors: CHEN, Lishan, DAUGHERTY, Sean Christopher, SVERDRUP, Francis Michael
• Format: Patent
• Language: English; French
• Published: 10.03.2022
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

This application relates to double-stranded small interfering RNAs that modulate DUX4 gene expression and describes methods of inhibiting DUX4 gene expression by contacting a cell with said double-stranded small interfering RNAs. The application further provides compositions comprising said double-stranded small interfering RNAs and their use in methods of preventing or treating a disease or disorder associated with aberrant expression of DUX4, such as facioscapulohumeral dystrophy (FSHD) or cancer, in a subject. Cette demande porte sur des petits ARN interférents double brin qui modulent l'expression du gène DUX4 et décrit des méthodes d'inhibition de l'expression du gène DUX4 par la mise en contact d'une cellule avec lesdits petits ARN interférents double brin. La demande concerne en outre des compositions comprenant lesdits petits ARN interférents double brin et leur utilisation dans des méthodes de prévention ou de traitement d'une maladie ou d'un trouble associé à une expression aberrante de DUX4, tel que la dystrophie facio-scapulo-humérale (FSHD) ou le cancer, chez un sujet.