Home / Health / A single shot of this novel drug blinds the patient.

A single shot of this novel drug blinds the patient.



International clinical trials are conducted through Perelman School of Medicine at the University of Pennsylvania helped childhood blindness gain vision after a single injection of RNA therapy into the eye.

The novel research may be published in a paper in Natural medicine On April 1

During the trial, the patient was given an intraocular injection shot into the eye of an oligonucleotide called sepofarsen, a small RNA molecule that targets the cone cells of the eye. It is responsible for color vision to increase the protein CEP290 levels and improve retinal function during the day.


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The possible treatment is aimed at patients with Leber congenital amaurosis (LCA), a rare and genetic eye disease that also affects the retina, and those with the CEP290 mutation.

Usually, patients with LCA are blind or severely visually impaired. Began in infancy

In this study, one patient was followed for 1

5 months after a single injection. The patient’s eyesight improved after a month, peaking in two months, and continued improvement even 15 months after a single injection.

“Our results set new standards for how biological improvements are possible,” said Artur Cideciyan, co-author of the trial and professor of ophthalmology research at Penn Medicine’s Scheie Eye Institute.

For the authors of the trial, including co-author Samuel Jacobson, the long duration of vision improvement after a single injection gave them hope that RNA therapy would have similar effects on other ciliopathies. Or a genetic mutation in the eye with a protein defect

“This work represents a very exciting direction for RNA antisense therapy. For 30 years, there are new drugs based on RNA antisense oligonucleotides. Even if everyone realizes there is a good promise for these treatments. ” Jacobson said.


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