NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING

• Main Authors: JOHNS RACHEL, LEVIN ARTHUR A, COCHRAN MICHAEL CARAMIAN, SHI YUNYU, DOPPALAPUDI VENKATA RAMANA, GEALL ANDREW JOHN, DARIMONT BEATRICE DIANA, BURKE ROB, HUANG HANHUA
• Format: Patent
• Language: Chinese; English
• Published: 13.10.2020
• 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; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

Disclosed herein are molecules and pharmaceutical compositions that induce an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exoninclusion. Also described herein include methods for treating a disease or disorder that comprises a molecule or a pharmaceutical composition that induces an insertion, deletion, duplication, or alteration in an incorrectly spliced mRNA transcript to induce exon skipping or exon inclusion. 本文公开了在错误剪接的mRNA转录物中诱导插入、缺失、复制或改变以诱导外显子跳读或外显子包含的分子和药物组合物。本文描述的还包括治疗疾病或病症的方法，其包括在错误剪接的mRNA转录物中诱导插入、缺失、复制或改变以诱导外显子跳读或外显子包含的分子或药物组合物。