Alternative to Using Viruses for Gene Therapy Breakthrough!

In a recent study conducted at Tufts University, researchers demonstrated that non-viral gene therapy can delay the onset of retinal degeneration of the eye and preserve vision. Small DNA carriers known as nanoparticles containing GDNF (Glial Cell Line-Derived Neutrophic Factor) were injected into the retinas of mice subjects and the responses were monitored. GDNF is a protein that protects the photoreceptor cells of the retina. Seven days after injection, the treated mice exhibited less retinal damage and better vision than the controls. However, the retinal protection provided by the treatment dwindled soon after, as no difference in vision between the two groups was present two weeks after treatment.

GDNF Protein expression

GDNF Protein expression

Previously, gene therapy has been carried out using viruses. Viruses are very efficient in delivering DNA to cells in vivo, but they can provoke immune responses that can lead to inflammation and other setbacks. Using nanopoarticles as part of non-viral gene therapy is a safer option that avoids these complications. The next step is to find ways to retain the retinal protection, as the ameliorative effects offered by the discussed treatment are currently short-lived.

Nevertheless, this study makes an important leap in finding ways to delay the onset of degenerative retinal diseases in humans, such as age-related macular degeneration and retinitis pigmentosa.

Brian Krawitz
Staff Writer

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