A single injection of this innovative drug reversed the patient’s blindness


An international clinical trial conducted through the The Perelman School of Medicine at the University of Pennsylvania helped a patient suffering from childhood blindness gain vision after a single injection of the experimental RNA therapy into the patient’s eye.

The potentially groundbreaking research was published in an article in Nature medicine April 1.

During the trial, patients received intraocular injections, injections into the eye, of an antisense oligonucleotide known as sepofarsen, a small RNA molecule that targets cells in the eye’s cone, which are responsible for color vision, to increase CEP290 protein levels and improve the functioning of the retina during the day.


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Possible treatment is aimed at patients with Leber’s congenital amaurosis (LCA), a rare genetic eye disorder that affects the retina, and who also have a CEP290 mutation.

In general, patients with ACL are blind or severely visually impaired. starting in childhood.

In this study, one of the patients was followed for 15 months after the single injection. The patient’s eyesight improved after one month, peaked in two months, and continued to improve even 15 months after the single injection.

“Our results set a new standard for potential biological enhancements,” said Artur Cideciyan, co-lead author of the trial and a research professor of ophthalmology at Penn Medicine’s Scheie Eye Institute.

For the trial authors, including co-lead author Samuel Jacobson, the long-term timeline of vision improvement after an injection gives them hope that RNA therapy will have similar impacts on other ocular ciliopathies or genetic mutations. with protein defections.

“This work represents a really exciting direction for RNA antisense therapy. It has been 30 years since new drugs that use antisense RNA oligonucleotides appeared, although everyone realized that these treatments were very promising. ” Jacobson said.


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