New technologies may prevent and reverse muscular dystrophy in mice
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Last Update: 2020-12-27
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Source: Internet
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Author: User
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a CRISPR-driven gene activation technique can prevent and reverse the symptoms of muscular dystrophy in mouse models. Muscular dystrophy is a kind of inherited muscle depletion disease, the cause of subtype congenital muscular dystrophy type 1A (MDC1A) is due to mutations in the Lama2 gene of coding layer adhesion protein alpha2, which causes some peripheral nerves to dislodge protective myelin and damage muscle fiber stability. Rodent studies have shown that increasing the expression of the associated gene Lama1 (coding layer adhesion protein alpha1) can help alleviate the symptoms of the mouse model of the disease, but because of the large Lama1 gene, standard gene therapy is difficult to achieve results.
to address this problem, Ronald Cohn and colleagues at Toronto Children's Hospital in Canada used a CRISPR-mediated gene activation system to increase the expression of layered adhesive protein alpha1 in the MDC1A mouse model. Mice that did not develop the condition were treated to prevent muscle depletion and paralysis;
CRISPR technology can be used to up or down the expression of key genes. In a study published online July 25 in Nature, the authors say that in the future, they may be able to "raise" protective genes and "lower" disease-caused genes through combination therapies to help treat the disease and other genetic disorders. (Source: Jin Nan, China Science Journal)
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