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A single shot restored the vision of blind children in one year



According to a new study published in the journal, patients with hereditary childhood blindness can recover and retain their vision for more than a year after receiving only a single experimental RNA treatment. Natural medicine.

Although impressive, it is important to encourage cautious optimism about this development-because there is no panacea for blindness.

One eye improves the central vision of blind children

This treatment is designed for people who have been pre-diagnosed with Leber Congenital Black Eye (LCA), an eye disease that usually affects the retina, and also has a CEP290 mutation, which is usually associated with patients with this disease . People with this type of LCA usually suffer severe visual impairment from infancy.

“Our results set a new standard for the biological improvement of LCA antisense oligonucleotide therapy caused by the CEP290 mutation.”

; MedicalXpress report. “Importantly, we have established a comparator for currently ongoing gene editing therapies for the same disease, which will allow to compare the relative advantages of two different interventions.”

Researchers at the Scheie Eye Institute at the Perelman School of Medicine at the University of Pennsylvania conducted this clinical trial and found that the treatment resulted in substantial changes in the fovea (a key part of human central vision). The Department of Medicine at the University of Pennsylvania is led by Samuel G. Jacobson, an international professor of ophthalmology.

During the study, an antisense oligonucleotide called sepofarsen was injected into the subject’s eye. This is a short RNA molecule that can increase the protein level of CEP290 in the photoreceptor cells of the eye and improve the function and perception of the retina under sunlight.

Improved vision maintained for more than 15 months

In a 2019 study Natural medicine, Jacobson, Cideciyan and other collaborators discovered how repeated Sepofarsen injections every three months improved the vision of 10 patients. The eleventh patient (whose treatment has been explained in a new paper in the same journal) received only one injection and was then examined over a 15-month period. Before treatment, the patient’s vision was reduced, his field of vision was narrow, and there was no night vision. However, after the injection, the scientists decided not to increase the quarterly maintenance dose, because doing so may cause cataracts.

After a single injection of Sepofarsen, scientists noticed a greater improvement, which fundamentally supported the biological effect of the therapy. In more than a dozen measurements of visual function and retinal structure, a key finding is that biological improvement is slow. The researchers recorded the improvement in vision after one month, but the patient’s vision peaked after two months.

The most important thing is that more than 15 months after a single injection, vision still has some improvement. This is unexpected, but it also has implications for the treatment of other ciliary diseases. Ciliopathy is a general disease associated with mutations in genes encoding abnormal proteins, which in turn causes abnormal function of cilia, which are sensory organelles that adhere to cells.

Jacobson said: “This work represents a truly exciting direction for RNA antisense therapy.” MedicalXpress report. “It has been more than 30 years. Although everyone is aware that the prospects for using these drugs are very optimistic, new drugs using RNA antisense oligonucleotides have appeared for 30 years. The ciliary transition zone noted in patients The unexpected stability of the drug also prompted people to reconsider Sepofarsen’s dosing regimen as other cilia treatments.

An important step towards new technology to help restore vision

Researchers believe that one of the reasons antisense oligonucleotides can improve vision is that tiny RNA molecules are small enough to sneak into the nucleus, but long enough to play a substantial role.

The chairman of the ophthalmology department said: “At least in the field of ophthalmology, there have been a series of clinical trials of antisense oligonucleotides for different genetic defects. These successes are the result of the success of Cide290yan and Dr. Jacobson in the CEP290-related LCA.” Joan O-Brien of Lehrman School of Medicine and the director of the Scheie Eye Institute.

Obviously, this is not a blind treatment. But this is still an important step in the development of new technologies that can fundamentally improve the direct low vision of people who are genetically prone to eye diseases.




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