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J Invest Dermatol. 2019 Jun;139(6):1221-1226. doi: 10.1016/j.jid.2018.11.036. Epub 2019 May 5.

Gene Therapy for Epidermolysis Bullosa.

Author information

1
Department of Dermatology, Program in Epithelial Biology, Stanford University School of Medicine, Stanford, California, USA; Department of Dermatology, Palo Alto Veterans Affairs Medical Center, Palo Alto, California, USA. Electronic address: mpm@stanford.edu.
2
Department of Dermatology, Program in Epithelial Biology, Stanford University School of Medicine, Stanford, California, USA.

Abstract

Epidermolysis bullosa is a family of diseases characterized by blistering and fragility of the skin in response to mechanical trauma. Advances in our understanding of epidermolysis bullosa pathophysiology have provided the necessary foundation for the first clinical trials of gene therapy for junctional and dystrophic epidermolysis bullosa. These therapies show that gene therapy is both safe and effective, with the potential to correct the molecular and clinical phenotype of patients with epidermolysis bullosa. Improvements in gene delivery and in preventing immune reactions will be among the challenges that lie ahead during further therapeutic development.

PMID:
31068252
DOI:
10.1016/j.jid.2018.11.036

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