Biomineralization nano material for treating Duchenne muscular dystrophy and gene editing system
Patent Information
- Authority / Receiving Office
- CN Β· China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- HUAZHONG AGRI UNIV
- Publication Date
- 2022-05-17
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Abstract
Description
technical field
[0001] The invention relates to a biomineralized nanomaterial and a gene editing system for treating Duchenne muscular dystrophy. Background technique
[0002] Duchenne muscular dystrophy, also known as pseudohypertrophic muscular dystrophy (DMD), is caused by mutations in the dystrophin gene (Dystrophin, Dys). DMD is a recessive genetic disease and the most common inherited muscle disorder in early childhood, with a prevalence of 1 / 5000 in males. The DMD gene encodes dystrophin, which is associated with the sarcolemma of cardiac and skeletal muscles and is critical for maintaining and supporting the structure and function of muscle tissue. Mutations in the DMD gene lead to loss of dystrophin expression, leading to severe muscle atrophy, myocardial failure, and respiratory failure in DMD patients, and most patients die before the age of 30.
[0003] Mutations in the DMD gene are primarily caused by exon deletions that lead to mutations in stop codons that d...