PMID: 9553721Apr 29, 1998Paper

Retrovirus-mediated DNA repair gene transfer into xeroderma pigmentosum cells: perspectives for a gene therapy

Cell Biology and Toxicology
L ZengM Mezzina

Abstract

The rare hereditary disease xeroderma pigmentosum (XP) is clinically characterized by extreme sun sensitivity and an increased predisposition for developing skin cancer. Cultured cells from XP patients exhibit hypersensitivity to ultraviolet (UV) radiation due to the defect in nucleotide excision repair (NER), and other cellular abnormalities. Seven genes identified in the classical XP forms, XPA to XPG, are involved in the NER pathway. In view of developing a strategy of gene therapy for XP, we devised recombinant retrovirus-carrying DNA repair genes for transfer and stable expression of these genes in cells from XP patients. Results showed that these retroviruses are efficient tools for transducing XP fibroblasts and correcting repair-defective cellular phenotypes by recovering normal UV survival, unscheduled DNA synthesis, and RNA synthesis after UV irradiation, and also other cellular abnormalities resulting from NER defects. These results imply that the first step of cellular gene therapy might be accomplished successfully.

Citations

Jun 26, 1999·Journal of Cutaneous Medicine and Surgery·A K SomaniK A Siminovitch
Dec 16, 2004·Proceedings of the National Academy of Sciences of the United States of America·Maria Carolina N MarchettoCarlos F M Menck
Nov 4, 2000·Expert Opinion on Investigational Drugs·W Pfützner, J C Vogel
May 31, 2019·Emerging Topics in Life Sciences·Gaetano Naso, Anastasia Petrova

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