Mice display improvements in vision following gene therapy

Japanese researchers have highlighted that 10% of photoreceptors were rescued using the pioneering approach

Blue DNA
Scientists have explored the effectiveness of replacing mutated genes in mice with congenital blindness.

Describing their findings in Nature Communications, researchers highlighted that replacing a mutated sequence in blind mice resulted in approximately 10% of photoreceptors being rescued.  

Following the procedure, the light sensitivity and visual acuity of the mice improved.

The new approach is an alternative strategy to gene supplementation, which has limitations when treating patients with defects in larger genes.

Koji Nishiguchi, from Tohoku University’s department of advanced ophthalmic medicine, explained that the new technique enables the replacement of a mutated sequence with its healthy counterpart.

“The platform paves the way for treating patients with mutations in larger genes, which comprise the vast majority of those with inherited retinal degeneration. Furthermore, a similar approach can be applied to treat almost any ocular and non-ocular inherited conditions,” he shared.

The research team are developing the genome editing platform for application in patients with retinitis pigmentosa. A clinical trial could be undertaken by 2025.

Image credit: Pixabay/Arek Socha