Improvement of muscular atrophy by AAV-SaCas9-mediated myostatin gene editing in aged mice.

ABSTRACT: Muscle mass and area usually decrease with age, and this phenomenon is known as sarcopenia. This age-related atrophy correlates with insufficient levels of muscle cells differentiate and proliferate regulated by the TGF-β signaling pathway and the expression of E3s ubiquitin-protein ligase by the aged. Sarcopenia makes a huge impact on the aging society, because it has the characteristic of high incidence, extensive adverse effects and disease aggravation gradually. Guided by a single-guide RNA (sgRNA), Cas9 nuclease has been widely used in genome editing, opening up a new pathway for sarcopenia treatment. Here, we present two rAAV9 systems, pX601-AAV-CMV:SaCas9-U6:sgRNA and pX601-AAV-EF1α:SaCas9-tRNA_GLN: sgRNA, which edited myostatin efficiently. By delivering the two rAAV-SaCa

SUBMITTER: Weng S 

PROVIDER: S-EPMC7725670 | biostudies-literature | 2020 Dec

REPOSITORIES: biostudies-literature