[Epidermal stem cells and ex vivo cutaneous gene therapy: application to xeroderma pigmentosum].
Ex vivo cutaneous gene therapy is an alternative treatment for recessively inherited diseases with cutaneous traits. It relies on the transfer in cultured epidermal keratinocytes of the wild-type allele of the gene whose mutation is responsible for the disease. As for severely burnt patients, epithelial sheets developed from genetically corrected cells may then be grafted back to the patients. Long term correction and graft take depend on the genetic correction of stem cells. Success of such an approach has recently been reported in the case of one patient suffering from a severe case of junctional epidermolysis bullosae. Here we report a method for safely selecting keratinocytes populations after genetic manipulation. The method is non invasive and non immunogenic and allows high enrichment of genetically manipulated stem keratinocytes. This could perhaps contribute to ex vivo gene therapy approaches of cancer prone genodermatoses such as xeroderma pigmentosum.
Warrick
Emilie
E
Génétique et physiopathologie des cancers épidermiques. Génomes et cancers, CNRS FRE 2939, Institut Gustave Roussy, 39, rue Camille Desmoulins, 94805 Villejuif, France.
Bergoglio
Valérie
V
Bernerd
Françoise
F
Magnaldo
Thierry
T
D000078404:Epidermal Cells; D004817:Epidermis / Q000502:physiology*; D015316:Genetic Therapy / Q000379:methods*; D018859:Hair Follicle / Q000166:cytology / Q000502:physiology; D006801:Humans; D009154:Mutation; D009381:Neoplasms, Radiation-Induced; D012878:Skin Neoplasms / Q000209:etiology; D013234:Stem Cells / Q000166:cytology* / Q000502:physiology*; D014466:Ultraviolet Rays / Q000009:adverse effects; D014983:Xeroderma Pigmentosum / Q000235:genetics* / Q000532:radiotherapy / Q000628:therapy*