Scheie Eye News Today

About 79% of clinical trial participants experienced measurable improvement after receiving experimental, CRISPR-based gene editing that is designed to fix a rare form of blindness, according to a paper published in the New England Journal of Medicine.

A recent clinical trial, BRILLIANCE demonstrated the safety and efficacy of CRISPR gene editing in treating inherited blindness caused by mutations in the CEP290 gene.

BRILLIANCE trial results showed 11 out of 14 treated participants experienced some improvements in vision and quality of life measures. CRISPR-based therapy

Alicia Gresham, senior vice president and COO of Mount Sinai Health System, has been named CEO of Pennsylvania Hospital, part of Philadelphia-based Penn Medicin

The years cohort of six recipients include a journalist, an astrophysicist, and Nobel Prize winners.

Mutation-Specific RNA Therapy Can Improve Vision with Lasting Effects April 5, 2021 A Penn Medicine patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye, according to researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. Results of the trial (“Durable vision improvement after a single treatment with antisense oligonu...

A Single Injection Reverses Blindness in Patient with Rare Genetic Disorder – Another RNA Success Apr 3, 2021 A patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye. Vanessa Bumbeers The gene editing research was conducted at the Perelman School of Medicine in the University of Pennsylvania. Results of the case, detailed in a paper published April 1 in Nature Medicine...

A Penn Medicine patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye.