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Reversing blindness in patients with rare hereditary diseases with a single injection – success of another RNA

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Patients with hereditary forms of childhood blindness acquired visual acuity that lasted more than a year after a single injection of RNA therapy into the eye.

Vanessa Bambiers

The genetic editing study was conducted at the Perelman School of Medicine at the University of Pennsylvania. The results of the case are detailed in a treatise published on April 1. Nature medicine, Indicates that the treatment has resulted in significant changes in the fovea, the most important point of human central vision.

In an international clinical trial, participants received an intraocular injection of an antisense oligonucleotide called sepophalsen. This short RNA molecule works by increasing normal CEP290 protein levels in the photoreceptors of the eye and improving retinal function under daytime visual conditions.

This treatment was designed for patients diagnosed with Leber congenital amaurosis (LCA), an eye disease that primarily affects the retina. The CEP290 mutation is one of the genes most often involved in patients with this disease. Patients with this form of LCA suffer from severe visual impairment, which usually begins in infancy.

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“Our results set a new standard that biological improvement is possible,” said Dr. Artur Cideciyan, co-author of the Ophthalmology Research Professor at the Scheie Eye Institute in Penn Medicine. “Importantly, we have established a comparator for ongoing gene editing therapies for the same disease, which allows us to compare the relative benefits of two different interventions.”

In the 2019 study published in Nature medicine, Cideciyan and Dr. Collaborators, including Samuel G. Jacobson, found that repeated injections of sepofalsen every three months resulted in continuous improvement in vision in 10 patients.

The eleventh patient, whose treatment details are described in the latest treatise, received only one injection and was tested for 15 months. Prior to treatment, the patient had poor eyesight, narrow vision, and no night vision. After the initial dose, patients decided to discontinue quarterly maintenance because regular doses can lead to cataracts.

Significant improvement in single “micro” dose

After a single injection of Sepofalsen, more than a dozen measurements of visual function and retinal structure showed significant improvements supporting the biological effects from treatment. An important finding from this case was that the uptake of this biological effect was relatively slow. Researchers saw an improvement in visual acuity after one month, but the patient’s visual acuity peaked after two months. Most striking is the improvement seen when tested for more than 15 months from the first and only injection.

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Prolonged persistence of visual improvement is unexpected and affects the treatment of other ciliopathy, according to researchers — the name of a large category of diseases associated with gene mutations that encode defective proteins. Found in cells, which results in the abnormal functioning of cilia, prominent sensory organelles.

“This study represents a very exciting direction for RNA antisense therapy. It’s been 30 years since new drugs using RNA antisense oligonucleotides appeared, and these therapies are big. Everyone was aware of the expectations, “said Jacobson. “The unexpected stability of the ciliary transition zone observed in patients encourages a review of sepophalsen dosing schedules and other ciliary target therapies.”

Researchers have shown that antisense oligonucleotides have proved successful in treating this rare disease because these small RNA molecules are small enough to enter the cell nucleus but are not removed immediately. ,work.

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For future research, Penn’s authors are planning gene-specific treatments for other currently incurable blinded hereditary retinal disorders.

Source: Pen medicine

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