Revertant mosaicism in skin: Natural gene therapy

Joey E. Lai-Cheong, John A. McGrath, Jouni Uitto

Research output: Contribution to journalReview articlepeer-review

103 Citations (Scopus)

Abstract

Revertant mosaicism is a naturally occurring phenomenon involving spontaneous correction of a pathogenic mutation in a somatic cell. Recent studies suggest that it is not a rare event and that it could be clinically relevant to phenotypic expression and patient treatment. Indeed, revertant cell therapy represents a potential 'natural gene therapy' because in vivo reversion obviates the need for further genetic correction. Revertant mosaicism has been observed in several inherited conditions, including epidermolysis bullosa, a heterogeneous group of blistering skin disorders. These diseases provide a useful model for studying revertant mosaicism because of the visual and accessible nature of skin. This overview highlights the latest developments in revertant mosaicism and the translational implications germane to heritable skin disorders.

Original languageEnglish
Pages (from-to)140-148
Number of pages9
JournalTrends in Molecular Medicine
Volume17
Issue number3
DOIs
Publication statusPublished - 2011 Mar

All Science Journal Classification (ASJC) codes

  • Molecular Medicine
  • Molecular Biology

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