SMaRT technology enables gene expression repair in skin gene therapy

J Invest Dermatol. 2008 Mar;128(3):499-500. doi: 10.1038/sj.jid.5701241.

Abstract

In this issue, Wally et al. (2008) report successful gene expression repair by spliceosome-mediated RNA trans-splicing (SMaRT), a novel achievement in molecular medicine. In their model, SMaRT was able to replace a mutation of the plectin gene in epidermolysis bullosa simplex with muscular dystrophy. This approach is particularly attractive for skin gene therapy of dominant-negative mutations present in a number of blistering genodermatoses.

Publication types

  • Comment
  • Review

MeSH terms

  • Epidermolysis Bullosa Simplex / genetics
  • Epidermolysis Bullosa Simplex / therapy*
  • Gene Expression
  • Genetic Therapy / methods*
  • Humans
  • RNA Splicing*