Ácidos nucleicos antisentido que inducen la omisión del exón 51

• Main Authors: HASEGAWA, Saki, HONDA, Yu, FUKUI, Takahiro, AOKI, Yoshitsugu, TAKEDA, Shin'ichi, MUCHIMA, Kaname
• Format: Patent
• Language: Spanish
• Published: 11.10.2022
• 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; 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

La presente memoria descriptiva proporciona un fármaco que provoca una omisión altamente eficaz del exón 51 en el gen de la distrofina humana. La presente memoria descriptiva proporciona un oligómero antisentido que tiene una actividad para inducir la omisión del exón 51 en el gen de la distrofina humana. The present specification provides a drug that causes highly-efficient skipping of exon 51 in the human dystrophin gene. The present specification provides an antisense oligomer having an activity to induce skipping of exon 51 in the human dystrophin gene.