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Scientists successfully test new way to deliver gene therapy​

Case Western Reserve University researchers use lipids to safely deliver gene therapy to the eye, successfully holding off advance of rare, inherited eye disorder
March 6, 2020
Zheng-Rong Lu

Zheng-Rong Lu. Credit Case Western Reserve University. 

Researchers at Case Western Reserve University have used a unique method to safely deliver gene therapy to fight a rare, but irreversible, genetic eye disorder known as Stargardt disease.

By using chemically modified lipids—instead of the viruses most commonly used as carriers—Stargardt disease did not return in animal models for up to eight months after treatment, said lead researcher Zheng-Rong Lu, the M. Frank Rudy and Margaret Domiter Rudy Professor of Biomedical Engineering at the Case School of Engineering, with a dual appointment at the School of Medicine.