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Home / Health / Single-dose treatment of this pioneering drug reversed the patient’s blindness

Single-dose treatment of this pioneering drug reversed the patient’s blindness



by The Perelman School of Medicine at the University of Pennsylvania helped patients with single-eye injections of experimental RNA therapy, allowing patients with childhood blindness to gain vision.

Potential breakthrough research published in Natural medicine On April 1.

During the trial, the patient received an intraocular injection of the antisense oligonucleotide Sepofarsen, a small RNA molecule that can target the cone cells of the eye (responsible for color vision) to increase CEP290 protein levels and improve the retina Features. That day.


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The potential treatment is for patients with Leber Congenital Black Eye (LCA), a rare hereditary eye disease of the retina, and CEP290 mutation.

Usually, LCA patients are blind or have severe visual impairment From infancy.

In this study, one of the patients was followed up for 1

5 months after a single injection. The patient’s vision improved after one month, reached a peak within two months, and continued to improve even 15 months after a single injection.

“Our results set a new standard for possible biological improvements,” said Artur Cideciyan, co-lead author of the trial and Professor of Ophthalmology at the Scheie Institute of Ophthalmology at Pennsylvania Medical University.

For trial authors, including co-lead author Samuel Jacobson (Samuel Jacobson), the long-term schedule of vision improvement after a single injection makes them hope that RNA therapy will treat other ciliated or protein-deficient eyes Genetic mutations have similar effects.

This work represents a truly exciting direction for RNA antisense therapy. It has been 30 years since the emergence of new drugs using RNA antisense oligonucleotides, although everyone realizes that these treatments have great promise,” Jacobson said.


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