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Satellite stem cells or myosatellite cells are quiescent stem cells that Alexander Mauro first identified in 1961. These cells are located between the sarcolemma, the plasma membrane of muscle fibers, and the basal lamina, the connective tissue sheath covering it. These mononucleated cells are activated in response to muscle injury, can transform into myoblasts, and may form or repair muscle fibers. Myosatellite cells can provide additional myonuclei for muscle regeneration or return to a...
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Alternative RNA splicing is the regulated splicing of exons and introns to produce different mature mRNAs from a single pre-mRNA. Unlike in constitutive splicing where a single gene produces a single type of mRNA, alternative splicing allows an organism to produce multiple proteins from a single gene and plays an important role in protein diversity.
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The Upf proteins that carry out nonsense-mediated decay (NMD) are found in all eukaryotic organisms, including humans. Each protein has an individual role, but they need to work in collaboration. Upf1 is an ATP-dependent RNA helicase that unwinds the RNA helix. Because Upf1 can unwind any RNA, Upf2 and Upf3 are required to help Upf1 discriminate between nonsense and normal mRNAs.
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Formation of Muscle Fibers from Myoblasts01:13

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De novo myogenesis, or the formation of muscle fibers, begins during the early embryonic stages. The skeletal muscle is formed from somites– blocks of embryonic cell layers. The somites are further divided into dermatomes, myotomes, sclerotomes, and syndetomes. Among these, the myotomes give rise to muscle fibers.
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Splicing is the process by which eukaryotic RNA is edited before its translation into protein. The RNA strand transcribed from eukaryotic DNA is called the primary transcript. The primary transcripts that become mRNAs are called precursor messenger RNAs (pre-mRNAs). Eukaryotic pre-mRNA contains alternating sequences of exons and introns. Exons are nucleotide sequences that code for proteins, whereas introns are the non-coding regions. In RNA splicing, introns are removed and exons are bonded...
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Translation01:31

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Translation is the process of synthesizing proteins from the genetic information carried by messenger RNA (mRNA). Following transcription, it constitutes the final step in the expression of genes. This process is carried out by ribosomes, complexes of protein and specialized RNA molecules. Ribosomes, transfer RNA (tRNA), and other proteins produce a chain of amino acids—the polypeptide—as the end product of translation.
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Corrigendum to 'Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen': Neuromuscular Disorders 31 (2021) 310-318/doi: 10.1016/j.nmd.2021.02.014.

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Updated: Mar 15, 2026

Multi-exon Skipping Using Cocktail Antisense Oligonucleotides in the Canine X-linked Muscular Dystrophy
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Noncoding RNAs and Duchenne muscular dystrophy.

Mark M Perry1, Francesco Muntoni1

  • 1The Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Institute of Child Health, 30 Guildford Street, London, WC1N 1EH, UK.

Epigenomics
|September 8, 2016
PubMed
Summary
This summary is machine-generated.

Noncoding RNAs (ncRNAs) show altered expression in Duchenne muscular dystrophy (DMD), potentially serving as biomarkers. Their therapeutic use in DMD requires further investigation.

Keywords:
Duchenne muscular dystrophyGRMD dogMDX micelncRNAmiRNA

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Area of Science:

  • Molecular Biology
  • Genetics
  • Biochemistry

Background:

  • Noncoding RNAs (ncRNAs), including microRNAs (miRNAs) and long noncoding RNAs (lncRNAs), regulate gene expression in response to various stimuli.
  • Evidence indicates ncRNAs are implicated in muscle pathologies, with a specific role emerging in Duchenne muscular dystrophy (DMD).

Purpose of the Study:

  • To review the current understanding of ncRNAs' role in Duchenne muscular dystrophy.
  • To explore the potential of ncRNAs as biomarkers for DMD.
  • To assess the feasibility of ncRNAs as therapeutic targets in DMD.

Main Methods:

  • Analysis of published studies investigating differential expression of miRNAs and lncRNAs in biological fluids from DMD patients and models.
  • Examination of potential mechanisms of action, including miRNA targeting by lncRNAs.
  • Review of findings related to fibrosis in DMD.

Main Results:

  • Differential expression of miRNAs has been observed in biological fluids of DMD patients and models (MDX mice, canine models) compared to controls.
  • Long noncoding RNAs are also differentially expressed in DMD patients, potentially influencing disease mechanisms by targeting miRNAs.
  • ncRNAs are linked to fibrosis, a common feature in DMD.

Conclusions:

  • ncRNAs are differentially expressed in Duchenne muscular dystrophy and may serve as valuable biomarkers for disease diagnosis and monitoring.
  • While ncRNAs show promise as biomarkers, their efficacy as therapeutic targets for DMD remains uncertain and requires further research.
  • Confirmation of recent findings is necessary to fully establish the role and utility of ncRNAs in DMD.