Long-Term Stability and Safety of Transgenic Cultured Epidermal Stem Cells in Gene Therapy of Junctional Epidermolysis Bullosa

Long-Term Stability and Safety of Transgenic Cultured Epidermal Stem Cells in Gene Therapy of Junctional Epidermolysis Bullosa

We report a long-term follow-up (6.5 years) of a phase I/II clinical trial envisaging the use of autologous genetically modified cultured epidermal stem cells for gene therapy of junctional epidermolysis bullosa, a devastating genetic skin disease. The critical goals of the trial were to evaluate th...

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Bibliographic Details
Main Authors: Laura De Rosa, Sonia Carulli, Fabienne Cocchiarella, Daniela Quaglino, Elena Enzo, Eleonora Franchini, Alberto Giannetti, Giorgio De Santis, Alessandra Recchia, Graziella Pellegrini, Michele De Luca
Format: Article
Language:English
Published: Elsevier 2014-01-01
Series:Stem Cell Reports
Online Access:http://www.sciencedirect.com/science/article/pii/S2213671113001264

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http://www.sciencedirect.com/science/article/pii/S2213671113001264

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