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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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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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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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Cell-Based Therapy for Spinal Muscular Atrophy.

Fabin Han1,2, Somayeh Ebrahimi-Barough3, Reyhaneh Abolghasemi3

  • 1The Institute for Translational Medicine, Shandong University/Affiliated Second Hospital, Jinan, Shandong, China. fhan2013@126.com.

Advances in Experimental Medicine and Biology
|October 26, 2020
PubMed
Summary

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by SMN gene mutations. Current treatments include nusinersen, and stem cell therapy shows promise for SMA treatment.

Keywords:
Motor neuronMutationSpinal muscular atrophyStem cellSurvival motor neuron (SMN)

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Area of Science:

  • Neuroscience
  • Genetics
  • Regenerative Medicine

Background:

  • Spinal muscular atrophy (SMA) is a severe neurodegenerative disease.
  • It results from mutations in the Survival Motor Neuron (SMN) gene.
  • SMA leads to motor neuron degeneration, progressive paralysis, and often early death.

Purpose of the Study:

  • To review the clinical, genetic, and pathogenic mechanisms of SMA.
  • To discuss current pharmacological treatments for SMA.
  • To explore the therapeutic potential of stem cell transplantation for SMA.

Main Methods:

  • Review of clinical and genetic data for SMA.
  • Analysis of pathogenic mechanisms underlying SMA.
  • Evaluation of current and emerging therapeutic strategies, including nusinersen and stem cell transplantation.

Main Results:

  • SMA is characterized by motor neuron loss due to SMN gene defects.
  • Nusinersen, an antisense oligonucleotide, is FDA-approved for SMA.
  • Stem cell transplantation demonstrates potential in preclinical SMA models.

Conclusions:

  • Understanding SMA's mechanisms is crucial for developing effective therapies.
  • SMN-targeted therapies like nusinersen offer treatment options.
  • Stem cell transplantation presents a promising future direction for SMA treatment.