A single epidermal stem cell strategy for safe ex vivo gene therapy. Issue 4 (27th February 2015)

• Record Type: Journal Article
• Authors: Droz‐Georget Lathion, Stéphanie; Rochat, Ariane; Knott, Graham; Recchia, Alessandra; Martinet, Danielle; Benmohammed, Sara; Grasset, Nicolas; Zaffalon, Andrea; Besuchet Schmutz, Nathalie; Savioz‐Dayer, Emmanuelle; Beckmann, Jacques Samuel; Rougemont, Jacques; Mavilio, Fulvio; Barrandon, Yann
• Is Part Of: EMBO molecular medicine. Volume 7:Issue 4(2015:Apr.)
• Page Start: 380
• Page End: 393
• Publication Date: 2015-02-27
• Subjects: Molecular biology -- Periodicals; Medical genetics -- Periodicals; Pathology, Molecular -- Periodicals; 616.04205
• DOI: 10.15252/emmm.201404353
• Languages: English
• ISSNs: 1757-4676

Abstract

<abstract abstract-type="graphical" xml:lang="en" id="emmm201404353-abs-0003"> <title> <x xml:space="preserve">Abstract</x> </title> <p>First time demonstration of a safe clonal strategy for <italic>ex vivo</italic> gene therapy before autologous transduced cells are transplanted into patients. Using recessive dystrophic epidermolysis bullosa (RDEB) COL7A1 corrected epidermal cloned stem cells as proof of principle, this strategy proves promising for clinical applications. <boxed-text content-type="graphic" id="emmm201404353-blkfxd-0002" position="anchor" orientation="portrait"><graphic position="anchor" mimetype="image" xlink:href="ark:/27927/pgjf84rzs2" orientation="portrait" xlink:type="simple" xmlns:xlink="http://www.w3.org/1999/xlink" /></boxed-text></p> </abstract>