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Updated: Aug 15, 2026

Generation and Culturing of Primary Human Keratinocytes from Adult Skin
10:42

Generation and Culturing of Primary Human Keratinocytes from Adult Skin

Published on: December 22, 2017

Gene therapy for autosomal dominant disorders of keratin

Alfred S Lewin1, Peter M Glazer, Leonard M Milstone

  • 1Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, Florida 32610-0266, USA. lewin@ufl.edu

The Journal of Investigative Dermatology. Symposium Proceedings
|October 28, 2005
PubMed

Insights

Genetic therapies for keratin diseases aim to suppress toxic protein production or correct genetic defects. Viral vectors offer a promising approach for long-term skin gene therapy, overcoming delivery challenges.

Area of Science:

  • Dermatology
  • Genetics
  • Molecular Biology

Background:

  • Dominant mutations in keratin genes cause severe epidermal diseases.
  • These mutations lead to non-functional keratin filaments, resulting in skin fragility and disease.

Purpose of the Study:

  • To explore genetic therapeutic strategies for keratin diseases.
  • To evaluate the potential of gene correction and RNA reduction for treating these conditions.

Main Methods:

  • Discusses gene correction in epidermal cells.
  • Explores RNA interference (RNAi) and other RNA reduction agents.
  • Highlights the challenge of skin impermeability for nucleotide delivery.
  • Proposes viral vectors for delivering gene expression inhibitors.

Main Results:

  • Gene correction is a viable goal for keratin diseases.
  • RNA reduction strategies are becoming plausible.
  • Skin impermeability poses a significant barrier to topical nucleotide delivery.
  • Viral vector-mediated gene delivery is an attractive alternative.

Conclusions:

  • Viral vectors offer a promising route for long-term genetic treatment of skin diseases caused by keratin mutations.
  • Overcoming skin barrier challenges is crucial for effective topical gene therapy.

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