Exploring Novel Gene Therapy Approaches to Treat Ocular Disease
Exploring Novel Gene Therapy Approaches to Treat Ocular Disease
Investigators find that nanoparticles deliver gene therapy successfully in mice, rats. Nanoparticles have been used to deliver gene therapy to treat age-related macular degeneration (AMD) in mice and rats. As reported in ScienceDaily, the Johns HopkinsUniversity (JHU) investigators used a uniquely engineered large molecule that facilitated compaction of large bundles of therapeutic DNA to be delivered into the ocular cells. This approach does not depend on viral vectors, as many of the gene therapies do, to transport the needed material into the cells. The downside of viral vectors is that an immune response is produced, and dosing cannot be repeated. In addition, the one used most often for ocular gene therapy cannot carry large genes, the investigators pointed out.
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