Irreversible vision loss may now not be a serious problem soon after scientists found a approach to alter the genes affecting different cells needed by the eyes to see. They managed to reverse vision loss in mice in a recent study.
Published within the Journal of Experimental Medicine on Friday, the study detailed how a team of researchers used a recent and highly versatile type of CRISPR-based gene editing to revive the vision of mice with retinitis pigmentosa.
Retinitis pigmentosa refers to a gaggle of rare eye diseases affecting the retina, particularly the light-sensitive photoreceptor cells. The rod and cone cells are answerable for sensing dim light and colours, respectively.
The genetic disease causes the breakdown of the retina cells, resulting in vision loss over time. Symptoms typically begin to surface in childhood, and other people lose their vision later in life. While there isn’t any cure for retinitis pigmentosa, vision aids and rehabilitation programs help patients profit from their remaining vision before irreversible vision loss takes place, in line with the National Eye Institute.
As one of the crucial common inherited diseases of the retina, retinitis pigmentosa affects 1 in 3,500 to 1 in 4,000 people in the USA and Europe, in line with data presented by MedlinePlus.
Previous research allowed scientists to revive vision in mice with other genetic diseases affecting the non-neuronal cells in the attention that support the rod and cone photoreceptor cells. The brand new study is different because it tackles essentially the most commonly inherited type of blindness affecting the neural photoreceptor themselves.
The team developed a flexible CRISPR system called PESpRY, which will be programmed to correct different genetic mutations occurring inside the genome. Retinitis pigmentosa is principally brought on by a mutation within the gene encoding a critical enzyme called PDE6β. By targeting the mutant gene, the system was in a position to restore the enzyme’s activity within the retinas of mice.
The researchers subjected the mice to behavioral tests to know if the technology saved the rod and cone photoreceptors of their eyes. The test animals found their way out of a visually guided maze water much like healthy mice. Additionally they responded well to visual stimuli.
“The flexibility to edit the genome of neural retinal cells, particularly unhealthy or dying photoreceptors, would offer far more convincing evidence for the potential applications of those genome-editing tools in treating diseases comparable to retinitis pigmentosa,” study creator Kai Yao, a professor on the Wuhan University of Science and Technology, said, as quoted by Neuroscience News.
Though the findings are very promising, Yao and colleagues admitted that much work continues to be needed to determine the protection and efficacy of the gene-editing tool in humans.
“Nonetheless, our study provides substantial evidence for the in vivo applicability of this recent genome-editing strategy and its potential in diverse research and therapeutic contexts, particularly for inherited retinal diseases comparable to retinitis pigmentosa,” Yao added.