COMPOSITION AND METHODS FOR MODULATING OF SMN2 SPLICING IN A SUBJECT

• Main Authors: KRAINER ADRIAN R, HUA YIMIN, SHIHABUDDIN LAMYA, KLINGER KATHERINE W, RIGO FRANK, BENNETT C. FRANK, PASSINI MARCO A, HUNG GENE, CHENG SENG H
• Format: Patent
• Language: English; Korean
• Published: 19.03.2024
• 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; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

본 명세서에는 대상에게서 SMN2 mRNA의 스플라이싱을 조정하는 화합물, 조성물 및 방법이 기술된다. 또한, 척수근위축증을 비롯한 질환 및 장애의 치료를 위한 약제의 제조에 있어 기술된 화합물 및 조성물의 용도를 제공한다. Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.