ANTISENSE COMPOUNDS AND METHODS FOR TARGETING CUG REDUCTIVES

• Main Authors: QIAN ZIQING, SHEN XIULONG, DOHERTY PATRICK, KELLARDI MARK, LI XIANG
• Format: Patent
• Language: Chinese; English
• Published: 30.04.2024
• 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; ORGANIC CHEMISTRY; PEPTIDES; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

Compounds comprising cyclic peptides, such as cyclic cell penetrating peptides, and antisense compounds are provided. The antisense compound binds to a gene having an amplified CTG repetitive sequence or a gene transcript having an amplified CUG repetitive sequence. The compounds may be delivered to a subject to treat diseases associated with amplified CTG.CUG repetitions, such as type 1 ankylosing muscular dystrophy (DMI), spinal cerebellar ataxial-8 (SCA8), and Huntington's Disease-2 (HDL2). 提供了包含环肽诸如环状细胞穿透肽及反义化合物的化合物。所述反义化合物与具有扩增的CTG重复序列的基因或具有扩增的CUG重复序列的基因转录物结合。所述化合物可递送至受试者以治疗与扩增的CTG·CUG重复序列相关的疾病，诸如1型强直性肌营养不良(DM1)、脊髓小脑性共济失调-8(SCA8)和亨廷顿病样-2(HDL2)。