Single stem cell gene therapy for genetic skin disease.
Stem cell gene therapy followed by transplantation into damaged regions of the skin has been successfully used to treat genetic skin blistering disorder. Usually, many stem cells are virally transduced to obtain a sufficient number of genetically corrected cells required for successful transplantation, as genetic insertion in every stem cell cannot be precisely defined. In this issue of EMBO Molecular Medicine, Droz-Georget Lathion et al developed a new strategy for ex vivo single cell gene therapy that allows extensive genomic and functional characterization of the genetically repaired individual cells before they can be used in clinical settings.
Université Libre de Bruxelles IRIBHM, Brussels, Belgium.
Jean-Christophe
JC
Larsimont
Université Libre de Bruxelles IRIBHM, Brussels, Belgium WELBIO Université Libre de Bruxelles, Brussels, Belgium.
Cédric
C
Blanpain
D000818:Animals; D024143:Collagen Type VII; D016108:Epidermolysis Bullosa Dystrophica / Q000628:therapy*; D005260:Female; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D008297:Male; D013234:Stem Cells / Q000378:metabolism*; D014161:Transduction, Genetic
D024143:Collagen Type VII