Bioscience News - GEG Tech top picks

The new generation CRISPR technology allows the restoration of the retinal and visual function after gene therapy in the treatment of a wide range of inherited ocular diseases. Using a mouse model with Leber Congenital Amaurosis (LCA) due to a clinically relevant pathogenic mutation in the Rpe65 gene, the team from the University of California at Irvine has successfully demonstrated the therapeutic potential of base editing for the treatment of LCA and by extension other blinding hereditary diseases. The researchers overcame some of the obstacles to the CRISPR-Cas9 system, such as unpredictable off-target mutations and low editing efficiency, by using cytosine and adenine base editors. The use of these editors allowed them to correct point mutations in a precise and predictable manner while minimizing unintentional mutations that could cause undesirable side effects. The basic editing treatment restored retinal and visual function in LCA mice to near normal levels. These results are extremely encouraging and represent a major step forward in the development of treatments for inherited retinal diseases.

A group of biotech veterans have debuted today a new company, Homology Medicine, with a bold claim that their underlying science is a better version of the gene editing methods, such as CRISPR-Cas9, that have captured the attention of patients, doctors, and scientists looking to treat desperate diseases.