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Genetic correction of stem cells in the treatment of inherited diseases and focus on xeroderma pigmentosum.
Int J Mol Sci. 2013 Oct 9;14(10):20019-36. doi: 10.3390/ijms141020019
Rouanet S, Warrick E, Gache Y, Scarzello S, Avril MF, Bernerd F, Magnaldo T
Abstract:
.....We have developed a safe procedure of genetic correction of epidermal stem cells isolated from XP patients. Preclinical and safety assessments indicate successful correction of XP epidermal stem cells in the long term and their capacity to regenerate a normal skin with full capacities of DNA repair.
PMID: 24113582
Free Full-Text: http://www.ncbi.nlm.nih.gov/pmc/articles/pmid/24113582/
Tags: gene therapy, methods, stem cells