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    Home > Biochemistry News > Biotechnology News > In gene editing specifically targeted muscle tissue MSTN treats evil diseases.

    In gene editing specifically targeted muscle tissue MSTN treats evil diseases.

    • Last Update: 2020-09-14
    • Source: Internet
    • Author: User
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    On December 5, the international academic journal Molecular Therapy published the latest research results of the Ding Qiuyu Research Group of the Institute of Nutritional Sciences of the Shanghai Institute of Life Sciences of the Chinese Academy of Sciences, Prevention of Muscle Wasting by CRISPR/Cas9-mediated Disruption of Myostatin In Vivo (click to read the original text in the lower left corner).
    the study, which looked at severe muscular dystrophy in tumor patients, suggested that the therapeutic purpose of delaying muscle atrophy was achieved by using CRISPR to target the removal of myostatin in muscle tissue.
    (cachexia) can be seen in a variety of diseases, of which tumor-related malincies are the most common.
    Tumor trophicity is a consumable syndrome shown by many cancer patients, especially in the late stages of the disease, which results in a significant loss of skeletal muscle (accompanied or not accompanied by a decrease in fat) despite adequate nutritional intake, leading to a serious decline in quality of life and survival.
    In this study, graduate student Wei Yuda and others, under the guidance of researcher Ding Qiuyu, used SaCRISPR/Cas9 to partially alleviate the evil disease by targeting myostatin in the body by using the myostatin signaling pathway activated in the wicked disease that induces muscle protein degradation.
    main reasons for choosing myostatin as the target are: 1) myostatin signaling path is a negative regulatory factor for muscle growth and development.
    have been found to carry myostatin functional deficiency mutation of natural persons, in addition to muscle system strength, clinically found no other serious adverse effects, targeted safety is guaranteed; 2) Pre-series of mice and clinical trials have shown that myostatin signaling pathlines play a key role in evil diseases, while monoantial resistance to myostatin is currently in clinical trials (treatment of virology in patients with pancreatic cancer); 3) myostatin is mainly secreted by muscle cells, the main action is self-secretion/side secretion, so the reduction of myostatin concentration in the local muscle micro-environment can retain part of the muscle function in the event of evil.
    based on this, Weyoda and others use muscle-specific promoter to initiate the expression of SaCas9 (staphylococcus aureus cas9), packaging into adeno-related virus vector (AAV), targeting mice Mstn with targeted injections of intestinal muscle tissue in mice Genes (coded myostatin protein), significant recovery of skeletal muscle function was observed in tumor-induced malignant mouse models (an average skeletal muscle weight increased by 9%, an average grip increased by 25%, and muscle fiber atrophy was significantly reduced by sliver staining at the injection point).
    study, as a validative experiment, suggests that the use of gene editing specifically targeted knock-out of myostatin in muscle tissue can be used as a potential gene therapy to prevent and treat evil diseases, while further improving the effectiveness of targeting in the body and comprehensively assessing target safety will contribute to its true clinical transformation.
    the study received support from nutrition institutes, Zhongshan Hospital, East China Normal University, as well as funding from the Chinese Academy of Sciences' 100-person program, the National Youth 1,000-person program and the Shanghai Pujiang Talent Program.
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