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Updated: Dec 10, 2025

Isolation and Culture of Adult Epithelial Stem Cells from Human Skin
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Isolation and Culture of Adult Epithelial Stem Cells from Human Skin

Published on: March 31, 2011

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Cellular therapy options for genetic skin disorders with a focus on recessive dystrophic epidermolysis bullosa

Gaetano Naso1, Anastasia Petrova1

  • 1Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health, 30 Guilford street, London WC1N 1EH, UK.

British Medical Bulletin
|September 5, 2020
PubMed
Abstract

Insights

Combinatorial cell and gene therapies show promise for recessive dystrophic epidermolysis bullosa (RDEB). Further research into stem cell applications and gene editing is needed for long-term clinical benefits in RDEB treatment.

Area of Science:

  • Regenerative Medicine
  • Dermatology
  • Genetic Disorders

Background:

  • Recessive dystrophic epidermolysis bullosa (RDEB) is a severe inherited skin disorder with limited treatment options.
  • Cell and gene therapies offer potential for RDEB treatment, with progress in preclinical and clinical studies.
  • Various cell types, including resident skin stem cells and adult stem cells, are being explored for RDEB therapy.

Purpose of the Study:

  • To review the current status and future directions of cell and gene therapies for RDEB.
  • To identify areas of agreement and controversy in RDEB cellular therapies.
  • To highlight promising research avenues for improving RDEB treatment outcomes.

Main Methods:

  • Literature review of recent publications.
  • Analysis of data from ClinicalTrials.gov and Clinicaltrialsregister.eu.
  • Review of pharmaceutical company press releases.

Main Results:

  • Autologous cell-based gene transfer shows positive outcomes, emphasizing the role of resident skin stem cells for long-term effects.
  • Adult stem cells may help systemic RDEB manifestations, but their contribution to skin regeneration is unclear.
  • Localized treatments have shown modest, transient improvements, while systemic therapies carry high risks.

Conclusions:

  • Combinatorial cell and gene therapies are essential for achieving long-term clinical benefits in RDEB.
  • Induced pluripotent stem cells and gene editing technologies present promising avenues for autologous RDEB therapies.
  • Further research is crucial to optimize cellular therapies and address current limitations in RDEB treatment.
Keywords:
MSCsRDEBcell therapygene therapyiPSCsskin cells

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