Therapeutic targets for modifying human dystrophin genes by gene editing and methods of use

• Main Authors: GERSBACH, CHRISTOPHER, A, ROBINSON-HAMM JAMES N
• Format: Patent
• Language: Chinese; English
• Published: 07.06.2024
• 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; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; VINEGAR; WINE

Disclosed herein are therapeutic targets for modifying human dystrophin genes by gene editing and methods of use. 本文披露了用于通过基因编辑修正人肌营养不良蛋白基因的治疗靶标和使用方法。