Gene editing halts vision loss in mice

Researchers prevent abnormal blood vessel growth in the retina


Scientists from the Schepens Eye Research Institute have prevented mice from developing angiogenesis of the retina using the gene-editing technique, CRISPR-Cas9.

Angiogenesis involves the abnormal growth of blood cells on the surface of the retina. It occurs in several degenerative eye conditions including proliferative diabetic retinopathy, wet age-related macular degeneration and retinopathy of prematurity.

As part of a study published in Nature Communications, scientists targeted a specific protein, VEGFR2, that is known to play an essential role in angiogenesis.

A viral vector was used to deliver genomic edits to the protein. Researchers found that a single injection of the gene therapy was able to prevent retinal angiogenesis in mice.

“This work establishes a strong foundation for genome editing as a strategy to treat angiogenesis-associated diseases,” the authors concluded.