Gene editing a potential approach to treat diabetic retinopathy and wet age-related macular degeneration

Using a gene editing technique, the CRISPR-Cas9 system, scientists from the Schepens Eye Research Institute have successfully prevented mouse models (which accurately mimic symptoms in human diseases) from developing angiogenesis of the retina.

Angiogenesis is a process which results in the growth of new blood vessels from pre-existing vessels in the body. An imbalance in angiogenesis can cause abnormal blood vessel growth, which is known to be a contributing factor to many conditions such as diabetic retinopathy and wet age-related macular degeneration.

During the study, scientists targeted a growth factor receptor called VEGFR2, which plays an important role in angiogenesis. In the study, angiogenesis in the mouse models was prevented using a single injection of the gene therapy.

Scientists believe that this research “establishes a strong foundation for genome editing as a strategy to treat angiogenesis-associated disease.” For more information read the published study in Nature Communications.