Article
Early intra-amniotic gene transfer using lentiviral vector improves skin blistering phenotype in a murine model of Herlitz junctional epidermolysis bullosa.
Gene therapy - 1 May 2012
Endo M, Zoltick P W, Radu A, Jiang Q, Qiujie J, Matsui C, Marinkovich P M, McGrath J, Tamai K, Uitto J, Flake A W
Abstract excerpt
Mutations of the LAMB3 gene cause a lethal form of junctional epidermolysis bullosa (JEB). We hypothesized that early intra-amniotic gene transfer in a severe murine model of JEB would improve or correct the skin phenotype. Time-dated fetuses from heterozygous LAMB3(IAP) breeding pairs underwent ultrasound guided intra-amniotic injection of lentiviral vector encoding the murine LAMB3 gene at embryonic day 8 (E8)....
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.