An international clinical trial conducted by 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 experimental RNA therapy into the patient’s eye.
The potentially groundbreaking research is in an article in Physical Medicine on April 1st.
During the trial, patients received intraocular injections, shots into the eye, of an antisense oligonucleotide known as sepofarsen, a small RNA molecule that targets the cone cells of the eye – responsible for color vision – to CEP290- increase protein levels and improve retinal function. the day.
America is changing faster than ever before! Add Changing America to you Facebook or Twitter feed to stay up to date with the news.
The possible treatment is aimed at patients suffering from congenital amaurosis (LCA), a rare and genetic eye disorder that affects the retina, and also has a CEP290 mutation.
Patients with LCA are usually blind or have a severe visual impairment start in infancy.
In this study, one of the patients was followed 15 months after the single injection. The patient’s vision improved after one month, peaked at two months, and continued to improve, even 15 months after the single injection.
“Our results set a new standard of biological enhancement that is possible,” said Artur Cideciyan, co-author of the dissertation and a research professor of ophthalmology at the Scheie Eye Institute of Penn Medicine.
For the authors of the trial, including co-lead author Samuel Jacobson, the long-term timeline of vision improvement after one injection gives them hope that RNA therapy will have similar impacts on other siliopathies or eye genetic mutations with protein disorders.
‘This work is a very exciting direction for RNA antisense therapy. “It’s been 30 years since there were new drugs that use RNA antisense oligonucleotides, although everyone realizes that there is a lot of promise for these treatments.” said Jacobson.
READ MORE STORIES ABOUT HEALTH AND MEDICINE
WHAT HAPPENS TO A DOSAGE OF PFIZER OR MODERN VACCINES?
OBAMACARE SIGNS 200K NEW ENTERPRISES
NEW PSYCHEDELICS STUDY PROPOSES ALSO PLACEBO WORK
IN A BREAKTHROUGH RESEARCHER IDENTIFIES CANCELS ON THEIR ACURITY
NEW BIOINK SCIENTISTS APPROACH TO PRESSURE HUMAN ORGANS