Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa

Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa

Abstract Background Recessive dystrophic epidermolysis bullosa (RDEB) is a rare, devastating blistering genodermatosis caused by mutations in the COL7A1 gene, which encodes for type VII collagen and is necessary for dermal-epidermal adhesion and integrity. Disease manifestations include severe and d...

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Bibliographic Details
Main Authors: Jodi Y. So, Jaron Nazaroff, Chinonso V. Iwummadu, Nicki Harris, Emily S. Gorell, Shivali Fulchand, Irene Bailey, Daniel McCarthy, Zurab Siprashvili, M. Peter Marinkovich, Jean Y. Tang, Albert S. Chiou
Format: Article
Language:English
Published: BMC 2022-10-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Epidermolysis bullosa
Recessive dystrophic epidermolysis bullosa
Genetic diseases
Genodermatoses
Blistering diseases
Gene therapy
Online Access:https://doi.org/10.1186/s13023-022-02546-9

Internet

https://doi.org/10.1186/s13023-022-02546-9

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