DHX36 / RHAU KNOCKOUT MICE AS EXPERIMENTAL MODELS OF MUSCULAR DYSTROPHY

摘要

The present invention provides a genetically-modified non-human animal whose somatic and germ cells contain a gene encoding an altered form of an DHX36 gene, the altered DHX36 gene having been targeted to replace a wild-type DHX36 gene into the animal or an ancestor of the animal at an embryonic stage using embryonic stem cells. An ideal use of the genetically-modified non-human animal of the invention is the use as an experimental model for muscular dystrophy, e.g. spinal muscular atrophy, to identify e.g. new treatments for muscular dystrophy and or study its pathogenesis.