AGENTES DE iARN PARA INHIBIR LA EXPRESIÓN DEL ENaC BETA, COMPOSICIONES DE ESTOS Y MÉTODOS DE USO

• Main Authors: Nicholas Anthony, Erik W. Bush, Casi M. Schienebeck, Xu Zhao, Li Zhen, Pei Tao, Zhu Rui
• Format: Patent
• Language: Spanish
• Published: 31.05.2021
• 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

Se describen agentes de iARN, composiciones que incluyen agentes de iARN y métodos para inhibición de un gen (SCNN1B) del ENaC beta. Los agentes de iARN del ENaC beta y los conjugados del agente de iARN que se divulgan en la presente inhiben la expresión de un gen del ENaC beta. También se describen composiciones farmacéuticas que incluyen uno o más agentes de iARN del ENaC beta, con opcionalmente uno o más agentes terapéuticos adicionales. La administración de los agentes de iARN del ENaC beta descritos a las células epiteliales, como ser, células epiteliales pulmonares. Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a beta-ENaC (SCNN1B) gene. The beta-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a beta-ENaC gene. Pharmaceutical compositions that include one or more beta-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described beta-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of beta-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including chronic obstructive pulmonary disease (COPD).