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The myogenic bHLH transcription factors--MyoD, Myf5, myogenin, and MRF4--control vertebrate skeletal muscle development (1). MyoD and Myf5 act redundantly as myoblast specification genes (2), whereas myogenin is required for myoblast [...], Members of the MyoD family of basic helix-loop-helix (bHLH) transcription factors control the formation of all skeletal muscles in vertebrates, but little is known of the molecules or mechanisms that confer unique identities to different types of skeletal muscles. MyoR and capsulin are related bHLH transcription factors expressed in specific facial muscle precursors. We show that specific facial muscles are missing in mice lacking both MyoR and capsulin, reflecting the absence of MyoD family gene expression and ablation of the corresponding myogenic lineages. These findings identify MyoR and capsulin as unique transcription factors for the development of specific head muscles.

The article discusses the restoration of dystrophin expression in duchenne muscular dsytrophy (DMD) using a single-cut genome editing. Topics mentioned include the contributing factor of deletions of exon 50 on DMD and the availability of various therapeutic strategies in restoring the DMD's muscle function like myoblast transfer. Also emphasized is the relevance of the single cut in genomic DNA in treating neuromuscular disorders and in clinically correcting DMD mutations.

The article discusses research into the prevention of Duchenne muscular dystrophy (DMD) in a mouse model through the editing of germline DNA. Topics include the use of clustered regularly interspaced short palindromic repeats/Cas9 (CRISPR/Cas9)-mediated genome editing, the correction of a mutation in the dystrophin gene (Dmd), and the potential implications of the study on future disease correction.

The article focuses on the Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the dystrophin gene (DMD). It mentions that high doses of adeno-associated virus (AAV) are required for efficient in vivo genome editing, posing challenges for clinical application; and also mentions the dose of scAAV required for efficient genome editing were at least 20-fold lower than with single-stranded AAV (ssAAV).

The article presents a study on CRISPR-Cas9. It outlines its treatment of Duchenne muscular dystrophy (DMD) exon 44 deletion mutations in human and mice cells. An overview of the significance of dosages of gene editing elements involving AAV9 and single guide RNA for genetic correction is also presented.

The article presents a study which uses genome editing with CRISPR/Cas9 to correct the diverse mutation in the human engineered heart muscle (EHM) of patients with Duchenne muscular dystrophy (DMD). Topics include the role of myoediting in correcting DMD mutations, the restoration of dystrophin protein expression in derivative cardiomyocytes, and the elimination of the genetic basis of cardiac abnormalities.

CRISPR/Cas9-mediated genome editing holds clinical potential for treating genetic diseases, such as Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene.To correctDMDby skippingmutant dystrophin exons in postnatalmuscle tissue in vivo,we used adeno-associated virus-9 (AAV9) to deliver gene-editing components to postnatal mdx mice, a model of DMD. Different modes of AAV9 delivery were systematically tested, including intraperitoneal at postnatal day 1 (P1), intramuscular at P12, and retro-orbital at P18. Each of thesemethods restored dystrophin protein expression in cardiac and skeletal muscle to varying degrees, and expression increased from 3 to 12 weeks after injection. Postnatal gene editing also enhanced skeletal muscle function, as measured by grip strength tests 4 weeks after injection.This method provides a potentialmeans of correctingmutations responsible for DMD and other monogenic disorders after birth. [ABSTRACT FROM AUTHOR]

Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene ( DMD ), is characterized by fatal degeneration of striated muscles. Dilated cardiomyopathy is one of the most common lethal features of the disease. We deployed Cpf1, a unique class 2 CRISPR (clustered regularly interspaced short palindromic repeats) effector, to correct DMD mutations in patient-derived induced pluripotent stem cells (iPSCs) and mdx mice, an animal model of DMD. Cpf1-mediated genomic editing of human iPSCs, either by skipping of an out-of-frame DMD exon or by correcting a nonsense mutation, restored dystrophin expression after differentiation to cardiomyocytes and enhanced contractile function. Similarly, pathophysiological hallmarks of muscular dystrophy were corrected in mdx mice following Cpf1-mediated germline editing. These findings are the first to show the efficiency of Cpf1-mediated correction of genetic mutations in human cells and an animal disease model and represent a significant step toward therapeutic translation of gene editing for correction of DMD.