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A revolutionary CRISPR gene editing technique applied for the first time to a patient with a visual disorder

16/03/20 - Leber Congenital Amaurosis, Gene editing, Therapy, Gene Therapy,

The patient, treated at the Casey Eye Institute in Portland, Oregon, belongs to a group of 18 people affected with Leber congenital amaurosis. These patients will participate in the BRILLIANCE study, the first clinical trial to determine the safety and efficacy of a therapy based on the CRISPR-Cas9 gene editing technique (AGN-151587, EDIT-101). It is the first example of an...

First gene therapy application of Luxturna in the UK

26/02/20 - Leber Congenital Amaurosis, Retinal Dystrophy, Therapy, Gene Therapy,

This February Moorfields Eye Hospital in London and Radcliffe Hospital in Oxford have started treating patients affected by Leber congenital amaurosis (LCA) with the Luxturna gene therapy. LCA is a childhood-onset severe retinal dystrophy, which causes irreversible blindness for which, until now, no treatment was available. Luxturna (voretigene neparvovec) is also the first gene therapy approved for the treatment of an...

Researchers develop a new non-invasive route to deliver gene therapy to the retina

25/10/19 - Macular degeneration, Therapy, Gene Therapy,

Researchers at the Johns Hopkins University have tested an alternative route to deliver gene therapy vectors to the retina that could potentially help patients with age-related macular degeneration (AMD) and inherited retinal disorders (IRDs). The new route of administration, called suprachoroidal injection, has been investigated initially in preclinical experiments in animals and uses a microneedle to inject gene-carrying therapeutic viruses...