Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa.

doi:10.1126/scitranslmed.3009540

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Article

Human COL7A1-corrected induced pluripotent stem cells for the treatment of recessive dystrophic epidermolysis bullosa.

Science translational medicine - 26 Nov 2014

Sebastiano Vittorio, Zhen Hanson Hui, Haddad Bahareh, Derafshi Bahareh Haddad, Bashkirova Elizaveta, Melo Sandra P, Wang Pei, Leung Thomas L, Siprashvili Zurab, Tichy Andrea, Li Jiang, Ameen Mohammed, Hawkins John, Lee Susie, Li Lingjie, Schwertschkow Aaron, Bauer Gerhard, Lisowski Leszek, Kay Mark A, Kim Seung K, Lane Alfred T, Wernig Marius, Oro Anthony E

Abstract excerpt

Patients with recessive dystrophic epidermolysis bullosa (RDEB) lack functional type VII collagen owing to mutations in the gene COL7A1 and suffer severe blistering and chronic wounds that ultimately lead to infection and development of lethal squamous cell carcinoma. The discovery of induced pluripotent stem cells (iPSCs) and the ability to edit the genome bring the possibility to provide definitive genetic...

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PMID: 25429056
DOI: 10.1126/scitranslmed.3009540
PMCID: PMC4428910

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