The first patients have been dosed in the phase 2/3 Sirius and Celeste clinical trials investigating QR-421a in patients with USH2A.

Coave Therapeutics enters into an exclusive licensing, co-development, and commercialization agreement with Théa Pharma.

Existing treatments only help a fraction of the estimated 100,000 Americans with this condition. But advances in gene therapy may soon help.

The aim of this review article is to give an overview of the current status of genotyping in ABCA4, an update on missing heritability

Mutations in pre-mRNA processing factors (PRPF3, 4, 6, 8, 31, SNRNP200, and RP9) have been linked to 15–20% of autosomal dominant RP cases.

X-linked RP caused by RPGR mutations, an attractive target for gene therapy because of its clinical severity and large number of patients.

Researchers developed and phenotyped a pigmented knockout rat model for lecithin retinol acyltransferase (LRAT) using CRISPR/Cas9.

Recessive mutations in DHDDS cause retinitis pigmentosa (RP59), resulting in blindness.

Here, we report 16 non-syndromic IRD patients and two cases with an IRD and some JBTS clinical features with pathogenic variants in INPP5E.

Consensus guidelines were developed to guide paediatricians and general ophthalmologists to arrive at the correct diagnosis of RPE65-associa

ProQR news – Positive results achieved in our ongoing Usher syndrome and retinitis pigmentosa research.

Ana B. Garcia-Delgado , Lourdes Valdes-Sanchez , Maria Jose Morillo-Sanchez , Beatriz Ponte-Zuñiga , Francisco J. Diaz-Corrales , Berta...

Before sharing a summary of our inaugural fundraiser, we again want to thank all who joined us on Saturday, April 24th. It was such a...

This review provides an overview of retinal gene therapy development by summarizing significant contributions and important clinical trials.

The FDA has granted orphan drug designation for chemically induced photoreceptor-like cells to treat retinitis pigmentosa.

RPE65-related LCA pivotal trials, . . . have paved the way for a new era of genetic treatments in ophthalmology.

By: Glenn Yiu, MD, PhD - Department of Ophthalmology & Vision Science, University of California, Davis, Sacramento, California Ophthalmology

A multi-center, chart review of 100 patients with RHO-associated adRP. Based on central visual fields, the optimal window of intervention is

The SNRNP200 gene plays a key role in the maturation of pre-mRNA splicing with the indication for the etiology of retinitis pigmentosa (RP).

CERKL mutations are an uncommon cause of arRP, but they are a significant cause of disease in populations with founder mutations