Recent advances in ex vivo gene therapy for xeroderma pigmentosum patients

Abstract

Perspectives of genetic treatment adapted to orphan monogenic genodermatosis have been fueled by tremendous progress in molecular genetics and gene transfer technologies. Xeroderma pigmentosum is a prototype of cancer prone syndromes; it is caused by severe alterations in nucleotide excision repair of DNA lesions introduced during exposure to sunlight. Here, the authors review the molecular basis of the disease and its impact on genotoxic insults caused by sunlight. The authors propose a compilation of preclinical results, revealing rational perspectives of cutaneous genetic correction of xeroderma pigmentosum with respect to appropriate specifications for this orphan and devastating disease.