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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Detalhes bibliográficos
Main Authors: De Rosa, Laura, Carulli, Sonia, Cocchiarella, Fabienne, Quaglino, Daniela, Enzo, Elena, Franchini, Eleonora, Giannetti, Alberto, De Santis, Giorgio, Recchia, Alessandra, Pellegrini, Graziella, De Luca, Michele
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2013
Assuntos:
Report
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3916757/
https://ncbi.nlm.nih.gov/pubmed/24511464
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2013.11.001
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