Single stem cell gene therapy for genetic skin disease

EMBO Molecular Medicine
Jean-Christophe Larsimont, Cédric Blanpain

Abstract

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.

References

Aug 16, 1984·The New England Journal of Medicine·G G GallicoH Green
Jun 15, 1999·Matrix Biology : Journal of the International Society for Matrix Biology·L Bruckner-TudermanN Hammami-Hauasli
Jul 10, 2009·The Journal of Clinical Investigation·Pierre A CoulombeElaine Fuchs
Jul 6, 2013·Regenerative Medicine·Sonia CarulliMichele De Luca
Nov 28, 2014·Science Translational Medicine·Vittorio SebastianoAnthony E Oro
Nov 28, 2014·Science Translational Medicine·Noriko Umegaki-AraoAngela M Christiano
Nov 28, 2014·Science Translational Medicine·Daniel WenzelJosef M Penninger
Mar 1, 2015·EMBO Molecular Medicine·Stéphanie Droz-Georget LathionYann Barrandon

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Citations

Dec 7, 2017·Journal of Applied Oral Science : Revista FOB·Camila Oliveira RodiniIan Campbell Mackenzie

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Methods Mentioned

BETA
biopsies
PCR

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