Correction of recessive dystrophic epidermolysis bullosa by transposon-mediated integration of COL7A1 in transplantable patient-derived primary keratinocytes

We report a gene delivery approach based on the Sleeping beauty transposon, which allows integration of a full-length COL7A1 cDNA and secretion of C7 at physiological levels in RDEB keratinocytes without rearrangements or detrimental effects on their clonogenic potential.

http://www.jidonline.org/article/S0022-202X(16)32888-3/fulltext?rss=yes

Source: Journal of Investigative Dermatology - December 23, 2016 Category: Dermatology Authors: Maria Carmela Latella, Fabienne Cocchiarella, Laura De Rosa, Giandomenico Turchiano, Manuel A.F.V. Gon çalves, Fernando Larcher, Michele De Luca, Alessandra Recchia Tags: Original Article Source Type: research