Chinese scientists find glimmer of hope for blind in gene-editing study
- Findings herald a potential breakthrough for millions affected by genetic eye diseases but cure is still some way off
- The researchers said they were able to precisely correct gene mutations in the retinal cells of mice
Around two million people worldwide suffer from inherited retinal diseases, the most common being Retinitis pigmentosa (RP), which causes progressive vision loss and eye disorders. There is no effective treatment.
But by correcting the genetic mutations that lead to the disorder, gene editing has the potential to restore normal protein expression and offer a lifelong cure, the researchers said.
Bi Changhao and Zhang Xueli, from the Chinese Academy of Sciences’ Tianjin Institute of Industrial Biotechnology, along with Wang Xiaolin and Sun Xiaodong from the National Clinical Research Centre for Eye Diseases, used gene editing to treat RP in mice.
According to the paper, traditional approaches to gene therapy – which involve injecting healthy copies of the affected gene into retinal cells – could not guarantee a balanced expression of the protein, and artificial DNA may degrade over time.
