Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use

• Main Authors: ROBINSON-HAMM JACQUELINE N, GERSBACH CHARLES A
• Format: Patent
• Language: Chinese; English
• Published: 09.11.2018
• Subjects: AGRICULTURE; ANIMAL HUSBANDRY; BEER; BIOCHEMISTRY; CARE OF BIRDS, FISHES, INSECTS; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; FISHING; FORESTRY; HUMAN NECESSITIES; HUNTING; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NEW BREEDS OF ANIMALS; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; SPIRITS; TRAPPING; VINEGAR; WINE

Disclosed herein are therapeutic targets for the correction of the human dystrophin gene 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。