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.
The United States is changing faster than ever!Add change to your Facebook or Twitter Feed to always follow the news.
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
“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.
Read more stories about health and medicine
What happens after a dose of modern or modern vaccination?
OBAMACARE attracts 200,000 new contracts
New psychological research shows that PLACEBO also has a lot of work
In a breakthrough researcher, the acid was used to identify cancer cells
New bioink scientists are closer to being able to print human organs