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Nonviral DNA nanoparticles for ocular gene therapy to treat Stargardt's disease

Mouse studies suggest nonviral DNA nanoparticles could be used for ocular gene therapy to help treat Stargardt's disease, a retinal degenerative disorder caused by mutations in ATP-binding cassette sub-family A member 4 (ABCA4; ABCR). In Abca4-deficient mice, nonviral DNA nanoparticles carrying human ABCA4 cDNA vectors and a photoreceptor-targeting promoter induced ABCA4 expression and decreased disease symptoms compared with saline. Next steps include testing the technology for delivery of a larger gene associated with vision disorders.

SciBX 5(34); doi:10.1038/scibx.2012.911
Published online Aug. 30, 2012

U.S. utility patent application filed; available for licensing

Han, Z. et al. J. Clin. Invest.; published online Aug. 13, 2012;
Contact: Muna I. Naash, The University of Oklahoma Health Sciences Center, Oklahoma City, Okla.