21 March 2023
Researchers in China have successfully restored the vision of mice with retinitis pigmentosa, one of the major causes of blindness in humans. BBC Science Focus reports:
Researchers in China have successfully restored the vision of mice with an inherited condition that leads to blindness.
The team, who are based at the Wuhan University of Science and Technology, used a newly developed CRISPR-based gene-editing technique to restore the sight of mice with retinitis pigmentosa.
The disease can be caused by mutations in more than 100 different genes. It causes photoreceptive cells in the retina to break down slowly over time, leading to vision loss and ultimately to blindness. It currently affects more than 1 in 4,000 people.
Gene-editing techniques have been used to restore vision in mice with genetic diseases, but only those that affect cells linked to the photoreceptors, not the photoreceptors themselves.
Vision restored in mice using revolutionary gene-editing technique https://t.co/xsX6uR6CQe
— BBC Science Focus Magazine (@sciencefocus) March 18, 2023
In @JExpMed, Qin, Zhang, Zhang, Feng, Yao et al. develop a genome-editing tool characterized by the versatility of prime editors and unconstrained PAM requirement of SpRY, and use the tool to rescue vision in a mouse model of retinitis pigmentosa. https://t.co/qyye9CWTJ0#CRISPR pic.twitter.com/WlFdQTUL2K
— Rockefeller U. Press (@RockUPress) March 17, 2023
The techniques used by the researchers could go beyond retinitis pigmentosa and could be a frontrunner in treating other genetic diseases.
“This is exciting evidence of the ability to correct blinding retinitis pigmentosa-like disorders in mice with a technique, which, hopefully, will evolve into human clinical trials,” said Dr. Howard R. Krauss, a surgical neuro-ophthalmologist and the director of Pacific Neuroscience Institute’s Eye, Ear & Skull Base Center at Providence Saint John’s Health Center in Callifornia.
“Having unlocked the molecular mysteries of genetic disorders decades ago, we have dreamed of reaching into the cell to add the missing genetic material,” he told Healthline. “There are now about 20 FDA-approved gene therapies for a variety of diseases. Yao and colleagues have made great strides in developing and demonstrating means of treating a wider variety of genetic retinal degenerative disorders.”
"The researchers indicated that much work must still be done to establish human safety and efficacy for these types of surgeries."
How CRISPR Gene Editing May Help Reverse Vision Loss https://t.co/ItOUPF2FPi via @healthline
— Church and State (@ChurchAndStateN) March 21, 2023
New gene-editing technique restores vision of blind mice
How Gene Editing is Transforming Our World
Imagining CRISPR Cures | Fyodor Urnov | TEDxBerkeley
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