Article
Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy.
Gene therapy - 1 Oct 2008
Kong J, Kim S-R, Binley K, Pata I, Doi K, Mannik J, Zernant-Rajang J, Kan O, Iqball S, Naylor S, Sparrow J R, Gouras P, Allikmets R
Abstract excerpt
Autosomal recessive Stargardt disease (STGD1) is a macular dystrophy caused by mutations in the ABCA4 (ABCR) gene. The disease phenotype that is most recognized in STGD1 patients, and also in the Abca4-/- mouse (a disease model), is lipofuscin accumulation in retinal pigment epithelium. Here, we tested whether delivery of the normal (wt) human ABCA4 gene to the subretinal space of the Abca4 -/- mice via...
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