Biomineralization nano material for treating Duchenne muscular dystrophy and gene editing system

A Duchenne muscle nutrition and gene editing technology, which is applied in the fields of biomineralized nanomaterials and gene editing systems to achieve the effects of high activity, fast and simple preparation method and low preparation cost
CN114504657APending Publication Date: 2022-05-17HUAZHONG AGRI UNIV

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

The invention discloses a biomineralization nanometer material for treating Duchenne muscular dystrophy and a gene editing system, the biomineralization nanometer material comprises the following components by volume: 1 part of carbonate, 2-5 parts of soluble calcium salt, 0.5 part of a polymer coating material, and 18-20 parts of a buffer solution; wherein the carbonate comprises, but is not limited to, Na2CO3 and K2CO3; the soluble calcium salt comprises but is not limited to CaCl2; the polymer coating material comprises but is not limited to PVP (Polyvinyl Pyrrolidone) and PAA (Polyacrylamide); the pH value of the buffer solution is 7-8, and the buffer solution does not contain carbonate; the biomineralized nano material can efficiently carry CRISPR / Cas9 plasmids, can protect the plasmids from being degraded in serum, and realizes gene editing in cells and organisms. The gene editing system provided by the invention adopts the biomineralized nano material as a basic carrier to carry a CRISPR / Cas9 plasmid system for treating Duchenne muscular dystrophy, and has wide application prospects and medical values.
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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...

Claims

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