[PDF][PDF] SMaRT technology enables gene expression repair in skin gene therapy.
UR Hengge - Journal of Investigative Dermatology, 2008 - researchgate.net
UR Hengge
Journal of Investigative Dermatology, 2008•researchgate.netIn 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.
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.
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.
researchgate.net