Xeroderma pigmentosum: from symptoms and genetics to gene-based skin therapy

Cells Tissues Organs. 2004;177(3):189-98. doi: 10.1159/000079993.

Abstract

Xeroderma pigmentosum (XP) is a rare, recessively inherited genodermatosis prone to ultraviolet (UV)-induced skin neoplasms from keratinocyte origin, i.e. basal and squamous cell carcinoma. Cells from classic XP patients fail to properly eliminate UV-induced DNA lesions by the nucleotide excision repair (NER) mechanism. A variant form of XP, called XP-V suffers from faulty translesion synthesis. We review here recent data on XP gene products whose alterations affect NER and result in one of the 7 complementation groups of XP. Encouraging results of retrovirus-based genetic correction of XP keratinocytes are summarized and support realistic prospects of gene therapy for the XP-C complementation group.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • DNA Damage / genetics
  • DNA Damage / radiation effects
  • DNA Repair / genetics
  • Genetic Therapy / methods*
  • Humans
  • Models, Biological
  • Skin / metabolism
  • Skin / radiation effects
  • Skin Neoplasms / prevention & control
  • Ultraviolet Rays / adverse effects
  • Xeroderma Pigmentosum / genetics*
  • Xeroderma Pigmentosum / metabolism
  • Xeroderma Pigmentosum / therapy*