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Related Concept Videos

Forced Transdifferentiation01:28

Forced Transdifferentiation

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Transdifferentiation, also known as lineage reprogramming, was first discovered by Selman and Kafatos in 1974 in silkmoths. They observed that the moths’ cuticle-producing cells transformed into salt-producing cells. Many such cases of natural transdifferentiation occur in organisms. In humans, pancreatic alpha cells can become beta cells. In newts, the loss of the eye’s lens causes the pigmented epithelial cells to transdifferentiate into the lens cells.
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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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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Related Experiment Video

Updated: Jan 16, 2026

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders
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Exploring Therapies for Duchenne Muscular Dystrophy Using Transdifferentiated Patient Fibroblasts.

Camila F Almeida1, Nicolas Wein2,3,4

  • 1Jerry R. Mendell Center for Gene Therapy, Abigail Wexner Research Institute, Nationwide Children's Hospital, Columbus, OH, USA.

Methods in Molecular Biology (Clifton, N.J.)
|October 1, 2025
PubMed
Summary

Duchenne muscular dystrophy (DMD) research can now use patient skin cells. This method converts skin fibroblasts into myogenic cells, enabling personalized therapy development for DMD and other neuromuscular disorders.

Keywords:
FibroblastMyoDMyoblastNeuromuscular disorderTherapy testingTransdifferentiationhTERT

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CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
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CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors

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Area of Science:

  • Biotechnology
  • Genetics
  • Cell Biology

Background:

  • Duchenne muscular dystrophy (DMD) results from numerous mutations in the DMD gene, causing dystrophin absence.
  • Current DMD treatments are not curative, partly due to the lack of patient-specific models for personalized therapy testing.
  • Existing animal models are insufficient to represent the vast spectrum of human DMD mutations.

Purpose of the Study:

  • To develop an efficient method for generating patient-derived myogenic cells from skin biopsies for DMD research.
  • To establish a reliable cell model for studying specific DMD mutations and screening novel biotherapies.
  • To overcome the limitations of primary myoblasts' proliferative capacity and the invasiveness of muscle biopsies.

Main Methods:

  • Fibroblasts from skin biopsies were immortalized using lentiviral transduction with hTERT.
  • A tetracycline-inducible MyoD construct was introduced via lentivirus for controlled differentiation.
  • Doxycycline addition triggered MyoD expression, inducing fibroblast transdifferentiation into myoblasts and then myotubes.

Main Results:

  • The protocol successfully transdifferentiated fibroblasts into functional myoblasts and subsequently into mature myotubes.
  • The resulting myotubes expressed DMD mRNA and key differentiation markers, including dystrophin.
  • This method provides a robust source of patient-specific myogenic cells from easily accessible skin biopsies.

Conclusions:

  • This rapid transdifferentiation protocol offers a valuable tool for investigating DMD pathogenesis and mutation effects.
  • The generated cell model facilitates the exploration of personalized gene-based and pharmacological therapies for DMD.
  • This approach holds promise for advancing research into DMD and other neuromuscular diseases.