Dystrophin gene editing by CRISPR/Cas9 system in human skeletal muscle cell line (HSkMC).

ABSTRACT:

Objectives

Duchene muscular dystrophy (DMD) is a progressive neuromuscular disease caused by mutations in the DMD gene, resulting in the absence of dystrophin expression leading to membrane fragility and myofibril necrosis in the muscle cells. Because of progressive weakness in the skeletal and cardiac muscles, premature death is inevitable. There is no curative treatment available for DMD. In recent years, advances in genetic engineering tools have made it possible to manipulate gene sequences and accurately modify disease-causing mutations. CRISPR/Cas9 technology is a promising tool for gene editing because of its ability to induce double-strand breaks in the DNA.

Materials and methods

In this study for the exon-skipping approach, we designed a new pair of guide RNA

SUBMITTER: Dara M 

PROVIDER: S-EPMC8591754 | biostudies-literature | 2021 Aug

REPOSITORIES: biostudies-literature