The joint battle to tackle epidermolysis bullosa through gene therapy
- PMID: 35599142
- DOI: 10.1016/j.molmed.2022.05.001
The joint battle to tackle epidermolysis bullosa through gene therapy
Abstract
Efforts are dedicated to definitively tackle skin lesions plaguing patients with epidermolysis bullosa (EB), a devastating genetic disorder affecting the integumentary system. Both in vivo gene therapy, as recently reported by Gurevich et al., and combined ex vivo cell and gene therapy strategies are under investigation. Here, we address the advantages and disadvantages of these different approaches.
Keywords: cell therapy; epidermal stem cells; epidermolysis bullosa; gene therapy; regenerative medicine.
Copyright © 2022 Elsevier Ltd. All rights reserved.
Conflict of interest statement
Declaration of interests M.D.L. is a co-founder and member of the Board of Directors of Holostem Terapie Avanzate, s.r.l., Modena, Italy.
Comment on
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In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial.Nat Med. 2022 Apr;28(4):780-788. doi: 10.1038/s41591-022-01737-y. Epub 2022 Mar 28. Nat Med. 2022. PMID: 35347281 Free PMC article. Clinical Trial.
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