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Lysosomes are the site for the degradation of macromolecules and biological polymers released during membrane trafficking events such as secretory, endocytic, autophagic, and phagocytic pathways. The membrane-enclosed area of the lysosome, called the lumen, contains hydrolytic enzymes active in an acidic environment. These acid hydrolases are functional at a pH between 4.5 and 5 and are involved in cellular processes such as cell signaling, energy metabolism, restoration of the plasma membrane,...
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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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Embryonic stem (ES) cells were first discovered in mice in 1981 by Martin Evans. In 1998, James Thomson identified a method to isolate embryonic stem cells from humans. Human embryonic stem cells (hESCs) are obtained from 3-5 day old embryos that remain unused after an in vitro fertilization procedure.
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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 Applications in Lysosomal Storage Disorders: Progress and Ongoing Challenges.

Sevil Köse1, Fatima Aerts-Kaya2,3, Duygu Uçkan Çetinkaya4,2

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Summary

Stem cell and gene therapies offer new hope for lysosomal storage disorders (LSDs). Combining these with existing treatments like enzyme replacement therapy shows promising results for patients.

Keywords:
Gene therapyHematopoietic stem cellInduced pluripotent stem cellLysosomal storage diseaseLysosomal storage disorderMesenchymal stem cellNeural stem cellStem cell

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Area of Science:

  • Biochemistry
  • Genetics
  • Cell Biology

Background:

  • Lysosomal storage disorders (LSDs) are rare genetic metabolic diseases.
  • Defective lysosomal function leads to substrate accumulation and cellular damage.
  • Current treatments like enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) have limitations.

Purpose of the Study:

  • To review the impact of LSDs on stem cell function.
  • To summarize stem cell-based therapies for LSDs.
  • To explore the potential of combining stem cell/gene therapy with existing treatments.

Main Methods:

  • Review of existing literature on LSDs and stem cell therapies.
  • Discussion of hematopoietic stem cells (HSCs), mesenchymal stem cells (MSCs), and induced pluripotent stem cells (iPSCs).
  • Analysis of advantages and disadvantages of different stem cell types and gene therapies.

Main Results:

  • LSDs affect stem cell function.
  • Stem cell-based therapies, including gene therapies, are under development for LSDs.
  • Combination therapies (stem cell/gene + ERT/SRT/chaperone) show promising results.

Conclusions:

  • Stem cell and gene therapies offer a hopeful future for LSD treatment.
  • Combination approaches may alleviate symptoms and prevent disease progression.
  • Further research into these combined therapies can improve patient outcomes.