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WHEN RARE IS RELATIVE
Although the IRD patient population is small compared with other retinal conditions, the research on novel therapeutic approaches is not.
Jul 31, 2024


Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial
Here, we report results of the first-in-human clinical trial (NCT04722107) of gene therapy for Bietti crystalline corneoretinal dystrophy
Apr 24, 2024


Preclinical dose response study shows NR2E3 can attenuate retinal degeneration in the retinitis pigmentosa mouse model RhoP23H+/−
Study demonstrates effectiveness of different doses of NR2E3 at reducing retinal degeneration and informs dose selection for clinical trials
Jan 26, 2024


Eluminex Biosciences Acquires Zuretinol Acetate from Retinagenix Holdings
Eluminex announced today it acquired rights for a novel oral therapy to treat LCA or RP caused mutations of the RPE65 or LRAT gene.
Jan 19, 2022


New Treatments for Retinitis Pigmentosa
Existing treatments only help a fraction of the estimated 100,000 Americans with this condition. But advances in gene therapy may soon help.
Aug 16, 2021


RPE65-associated inherited retinal diseases: consensus recommendations for eligibility to gene therapy
Consensus guidelines were developed to guide paediatricians and general ophthalmologists to arrive at the correct diagnosis of RPE65-associa
Jun 4, 2021


Dissecting the role of EYS in retinal degeneration: clinical and molecular aspects and its implications for future therapy
Ana B. Garcia-Delgado , Lourdes Valdes-Sanchez , Maria Jose Morillo-Sanchez , Beatriz Ponte-Zuñiga , Francisco J. Diaz-Corrales , Berta...
May 17, 2021


An Update on Gene Therapy for Inherited Retinal Dystrophy: Experience in Leber Congenital Amaurosis
This review provides an overview of retinal gene therapy development by summarizing significant contributions and important clinical trials.
Apr 26, 2021


LCA, early-onset severe retinal dystrophy: current management and clinical trials
RPE65-related LCA pivotal trials, . . . have paved the way for a new era of genetic treatments in ophthalmology.
Mar 12, 2021


Genome Editing in Retinal Diseases using CRISPR Technology
By: Glenn Yiu, MD, PhD - Department of Ophthalmology & Vision Science, University of California, Davis, Sacramento, California
Ophthalmology
Mar 7, 2021


Advanced late-onset retinitis pigmentosa with dominant-acting D477G RPE65 mutation is responsive to oral synthetic retinoid therapy
Objective: assess whether in a proof-of-concept study, oral synthetic 9 cis-retinyl acetate therapy improves vision in such advanced disease
Dec 1, 2020


Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence
Subretinal gene therapy trials began with the discovery of RPE65 variants and their association with Leber congenital amaurosis.
Nov 13, 2020


Research Models and Gene Augmentation Therapy for CRB1 Retinal Dystrophies
Discuss the recent advances, advantages and disadvantages of different CRB1 human and animal retinal degeneration models.
Aug 14, 2020


Biogen boosts gene therapy strategy with Harvard pact focused on inherited eye disease
Mutations in PRPF31 cause many more cases of RP than do mutations in RPE65, the gene targeted by Spark Tx Luxturna, Harvard researchers say.
Jul 6, 2020


New gene therapy technique
Shows promise in stem cell model of retinitis pigmentosa Although the 2017 FDA approval of Spark Therapeutics’ gene therapy Luxturna was...
Jun 10, 2020


Nr2e3 is a genetic modifier that rescues retinal degeneration and promotes homeostasis in multiple models of retinitis pigmentosa
Sujun Li , Shyamtanu Datta , Emily Brabbit , Zoe Love , Victoria Woytowicz , Kyle Flattery , Jessica Capri , Katie Yao , Siqi Wu ,...
Mar 2, 2020


Gene therapy to halt rare form of sight loss
The gene therapy is for patients who have retinal dystrophy as a result of inheriting a faulty copy of the RPE65 gene from both parents.
Feb 16, 2020


The effect of human gene therapy for RPE65-associated Leber’s congenital amaurosis on visual function: a systematic review and meta-analysis
This study aims to evaluate the association between changes in visual function and application of gene therapy in patients with RPE65-LCA.
Feb 14, 2020


First Gene Therapy FDA-Approved for an Inherited Retinal Disease
In 1972, Friedman and Roblin proposed that it was theoretically possible to introduce “good” DNA to replace defective DNA.
Apr 1, 2018


FDA Approves Gene Therapy for Inherited Blindness Developed by the UPenn and CHOP
In 2017, the FDA approved a gene therapy for RP caused by RPE65. The decision marked the first approved treatment for any form RP.
Dec 19, 2017
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