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The CRISPR Gene Editing Technique reverses vision loss in mice
Researchers were able to restore vision in mice suffering from retinitis pigmentosa using a versatile form of CRISPR gene edit.
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The study, which will be published in the Journal of Experimental Medicine on March 17, uses a novel, versatile form of CRISPR-based genome edit that has the potential to correct many disease-causing genetic mutations.
Genome editing has been used to restore vision in mice with genetic diseases such as Leber congenital aurosis. This is a layer of nonneuronal cells that supports the light-sensing rods and cone photoreceptors. Most inherited forms, such as retinitis pigmentosa and retinitis verifida, are caused by genetic defects in the neural photosensors.
Kai Yao, a professor at Wuhan University of Science and Technology, says that the ability to edit the DNA of neural retinal cell genomes, especially those with dying photoreceptors would be much stronger evidence for the potential uses of these genome-editing tools in the treatment of diseases like retinitis pigmentosa.
Mutations in more than 100 genes can cause retinal pigmentosa. It is estimated that 1 in 4000 people will experience vision impairment due to it. It starts with the death and dysfunction of dimly-sensing rod cells. Then it spreads to the cone cells that are required for color vision. Eventually, it can lead to irreversible vision loss.
Yao and his colleagues tried to save the vision of mice suffering from retinitis pigmentosa, which is caused by a mutation at the gene that codes a critical enzyme called PDE6b. Yao's team created a new CRISPR system called PESPRY that can be programmed to correct any type of genetic mutation regardless of their location in the genome.
The PESpRY system, when programmed to target mutant PDE6b genes, was able to effectively correct the mutation and restore enzyme activity in the retinas. This prevented the death of rod and cone photoreceptors and restored their normal electrical reactions to light.
Yao and his colleagues conducted a variety of behavioral tests to verify that the gene-edited mice still had good vision. The animals were able, for example, to navigate a visually-guided water maze just as well as healthy mice. They also showed typical head movements in response to visual stimuli.
Yao states, "However, our study provides substantial support for the in-vivo applicability and its potential in diverse therapeutic contexts and research, in particular for inherited retinal disorders such as retinitis pigmentosa."
After the mutation was corrected, the progressive cell loss was reversed. This resulted in the significant rescue of photoreceptors as well as the production of functional PDE6b. The mice that were treated showed significant electroretinogram responses and performed well in passive and active avoidance testing.
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