Gene Therapy Could Cure Blindness


Scientists in Oklahoma have published new research that suggests that intensive nanoparticle gene therapy could be used to develop a cure for blindness. The research, published today in the April edition of The FASEB Journal, details how scientists have successfully used gene therapy to restore the vision of blind mice. It is hoped that this research can be used to develop a cure for inherited retinal diseases including retinitis pigmentosa, macular degeneration and more.

Researchers conducting the study used mice that carried the gene proven to cause retinitis pigmentosa - an inherited retinal disease that affects the structure of the eye and causes vision to deteriorate over time. The team successfully treated the mice by using gene therapy and compacted synthetic DNA nanoparticles. They found that mice were able to see better after being exposed to this form of gene therapy. In fact, the research seems to show that using nanoparticle gene therapy has resulted in structural, functional and bio-chemical improvements to the eye.

The scientists behind this research found that using nanoparticles caused the structure of the retina, itself, to improve dramatically without causing any negative side effects. This has given hope that nanoparticle gene therapy could be used in future to successfully repair structural damage caused to the human eye by inherited diseases including retinitis pigmentosa. Retinitis pigmentosa is currently incurable and is thought to affect 1 in every 3000 people worldwide.

2nd April 2010

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