알파-시누클레인을 표적화하는 안티센스 올리고뉴클레오티드 및 그의 용도

• Main Authors: JENSEN MARIANNE LERBECH, CACACE ANGELA M, MCDONALD IVAR M, HAGEDORN PETER, DEVIDZE NINO, LOY JAMES K, BALDICK CARL J, PENDRI ANNAPURNA, OLSON RICHARD E, MEREDITH JERE E. JR
• Format: Patent
• Language: Korean
• Published: 16.12.2020
• 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; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

본 개시내용은 세포에서 SNCA mRNA를 (예를 들어, 인트론 엑손 연접부에서) 표적화하여, SNCA 단백질의 감소된 발현을 발생시키는 안티센스 올리고뉴클레오티드에 관한 것이다. SNCA 단백질 발현의 감소는 특정 의학적 장애, 예를 들어, 신경학적 장애의 치료에 유익하다. The present disclosure relates to antisense oligonucleotides, which target SNCA mRNA (e.g., at an intron exon junction) in a cell, leading to reduced expression of SNCA protein. Reduction of SNCA protein expression is beneficial for the treatment of certain medical disorders, e.g., a neurological disorder.