SMaRT technology enables gene expression repair in skin gene therapy.
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.
Hengge
Ulrich R
UR
Department of Dermatology, Heinrich-Heine University, Düsseldorf, Germany. ulrich.hengge@uni-duesseldorf.de
D016110:Epidermolysis Bullosa Simplex / Q000235:genetics / Q000628:therapy*; D015870:Gene Expression; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D012326:RNA Splicing