Gene therapy improves vision in blind mice


Thursday, 13 February, 2020


Gene therapy improves vision in blind mice

Mice born blind have shown significant improvement in vision after undergoing a new gene therapy, developed by scientists from Tohoku University and described in the journal Nature Communications.

The new method is an alternative strategy of gene supplementation, which involves supplementing the defective gene, such as the ones that can lead to inherited retinal degeneration, with a healthy one. The healthy gene is delivered via the adeno-associated virus (AAV). However, the virus can only hold a small healthy gene, and the vast majority of patients with defects in a larger gene cannot be treated with this method.

“To overcome this problem, we developed a single AAV gene therapy platform that allows local replacement of a mutated sequence with its healthy counterpart that can treat almost any mutation,” said Associate Professor Koji Nishiguchi, co-first author on the paper.

The single AAV gene therapy platform combines CRISPR-Cas9 technology with micro-homology-mediated end joining. These two things act essentially as genetic scissors and genetic glue respectively. Researchers can target a specific defective gene, cut it out and glue in a healthy replacement.

In blind mice, this approach rescued approximately 10% of photoreceptors, resulting in improved light sensitivity and an increase in visual activity. The improvement in vision was about the same result gene supplementation can produce.

“By treating mice blinded by inherited retinal degeneration with the new treatment, we show that a robust visual restoration can be achieved at a level similar to that mediated by conventional gene supplementation, assuring its potential for clinical application,” Nishiguchi said.

“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.”

The researchers are now applying the new genome editing platform to develop a therapy for human patients with retinitis pigmentosa, a group of rare conditions that can cause loss of peripheral vision and difficulty seeing at night. They will target common mutations among patients that remain untreatable by conventional gene therapy, and hope to have therapy in a clinical trial by as early as 2025.

Image credit: ©stock.adobe.com/au/oni

Please follow us and share on Twitter and Facebook. You can also subscribe for FREE to our weekly newsletters and bimonthly magazine.

Related News

Probiotics found to alleviate chronic stress in mice

A recent study took a systematic approach to test the efficacy of 12 candidate probiotics strains...

Baby born from egg matured in the lab and frozen

Fertility doctors have announced what is claimed to be the first baby to be born from an immature...

Antimicrobials found in sheep poo and on human skin

The newly discovered antimicrobials fall into a class of small antimicrobial proteins called...


  • All content Copyright © 2020 Westwick-Farrow Pty Ltd