Chinese scientists use CRISPR gene editing to restore sight in blind mice research Published on Friday in Journal of Experimental MedicineAnother advance that hints at potential applications in humans as scientists grapple with thorny ethical issues while taking up revolutionary technology.
Scientists successfully reverse vision loss in mice with retinitis pigmentosa, which is an inherited genetic disorder Situation It causes the cells in the retina to slowly break down over time and is one of the leading causes of blindness worldwide.
To do this, the scientists said they developed a new Crispr tool and used it to correct the genetic mutation responsible for the loss of vision.
The mice retained their vision even into old age, said the researchers, who assessed their visual skills using a variety of behavioral tests, including their ability to escape an aqueous maze.
However, the CRISP technology has been used to restore sight to blind mice, said Kai Yao, one of the researchers involved in the study and a professor at the Wuhan University of Science and Technology in China. forbes The tools used will not be able to efficiently correct many genetic causes of blindness in humans, including retinitis pigmentosa.
Yao said the more versatile Crispr tool developed by him and his colleagues is “capable of making any type of edit at any location in the genome” and “more convincing evidence” for a diverse array of gene editing’s potential uses for therapy. provides. Disease.
“This may be one of the most powerful genome editing tools available,” he added.
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"I'm excited by the results of the study," Yao said. forbes He and other researchers are keen to test the tool against other types of retinitis pigmentosa, which can be triggered by mutations in more than 100 different genes. However, human applications are still a long way off. "A lot of work still needs to be done to address safety concerns and ensure efficacy before this system can be used safely and effectively in humans," explained Yao.
Crispr, short for Clustered Regular Interspaced Short Palindromic Repeats, is a powerful gene-editing tool and one of the most admired innovations in modern biology. Derived from the bacterial immune system and often likened to a pair of genetic scissors, the Crispr system allows scientists to precisely alter DNA at specific locations. In theory, its uses are virtually limitless and in less than a decade it has transformed the life sciences. It has already been widely deployed in fields such as medicine and agriculture to treat, cure and prevent diseases, boost production, and even alive extinct animals. it's already happening testing in humans and—much to the international uproar—are used to make First Gene-edited babies.
1 in 4,000. The same number of people in the United States are affected by retinitis pigmentosa, research Estimate. The disease is also known as hereditary retinal dystrophy.
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Beyond CRISPR babies: how human genome editing is moving forward after the scandal (Nature)
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