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Research & Discovery 

Technical Notes

Functional Retroviral Vector for Gene Therapy of Xeroderma Pigmentosum Group D Patients

by M. Carreau, X. Quillet, E.Eveno, A. Salvetti, O. Danos, J-M. Heard, Alain Sarasin, et al.
Human Gene Therapy 6:1307-1315 (Oct. 1995)
Mary Ann Liebert, Inc.


Overview Summary


The first step toward gene therapy for the DNA repair disease xeroderma pigmentosum (XP) has been established by Carreau et al. They report the construction of a retroviral vector containing one of the DNA repair genes, the XP complementation group D previously known as ERCC2. Gene transfer was carried out on fibroblasts from two XP complementation group D patients referred to their laboratory. Complete phenotypic correction of the DNA repair defect in cells from both patients was achieved after the introduction of a functional copy of the XPD gene. This vector will then be used to modify keratinocytes to produce repair proficient reconstituted skin for engraftment at the most exposed areas. In this view, this study represents the first step toward long-term skin cancer therapy for XP patients.

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