19.10.2017

Boston Scientists Discover New Gene Therapy For Retinal Angiogenesis

Eye researchers in Massachusetts reversed a serious retinal disease using a new gene therapy procedure. Although researchers used lab mice in this study, doctors are hopeful they can use this same technique on humans who have diseases related to retinal angiogenesis.

Researchers at Schepens Eye Research Institute were primarily interested in getting rid of a protein that goes by the name of VEGFR2. This protein is a major contributor to angiogenesis.

The study's authors said they used a complex gene therapy technique known as CRISPR-Cas9 to help reduce the amount of VEGFR2 in the eye. These scientists actually sent in a viral strain to change the protein with genomic edits. It only took one injection of this serum to dramatically improve the mice's resistance to retinal angiogenesis.

For those who don't know, retinal angiogenesis refers to an unnatural overgrowth of blood vessels on the topmost layer of the retina. Just a few eye diseases related to angiogenesis include wet age-related macular degeneration (AMD) and proliferative diabetic retinopathy.

Both diabetic retinopathy and wet AMD can lead to blindness if doctors don't notice the signs early enough. These diseases don't present visual symptoms until they have advanced a great deal.

While there's no cure, doctors can slow down the progression of symptoms for AMD and diabetic retinopathy. Everyone over the age of 40 should get a yearly eye exam to check for these and other serious eye diseases.

In the official research paper, researchers wrote that this therapy could help reduce the incidence for angiogenesis-related eye diseases. They also said they hope their study will serve as a foundation for future research into CRISPR-Cas9 therapy.

Scientists at the Schepens Eye Research Institute research novel ways to treat and prevent blinding diseases. Founded in 1950 by retinal surgeon Charles Schepens, this research center has published well over 4,500 articles in prestigious medical journals. You can find this Harvard-affiliated research center at 20 Staniford Street in Boston.

Anyone interested in reading more about this study should check out the latest issue of Nature Communications. A few of the key authors on this study include professors Xionggao Huang, Guohong Zhou, and Patricia A. D'Amore. The title of this study is "Genome editing abrogates angiogenesis in vivo."


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