Leber Congenital Amaurosis News and Research

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Gut bacteria may be responsible for sight loss in certain inherited eye diseases

Sight loss in certain inherited eye diseases may be caused by gut bacteria, and is potentially treatable by antimicrobials, finds a new study in mice co-led by a UCL and Moorfields researcher.

26 Feb 2024

Engineered virus-like particles power up gene editing, correcting blindness in mice

Prime editing, a versatile form of gene editing that can correct most known disease-causing genetic mutations, now has a new vehicle to deliver its machinery into cells in living animals.

8 Jan 2024

Can genome editing transform ocular disease treatment?

The discovery and development of gene-editing technologies and their applications in managing ocular diseases.

3 Aug 2023

New agreement to facilitate genetic diagnosis of hereditary retinal dystrophies

An agreement between the University of Barcelona spin-off DBGen Ocular Genomics and the company Novartis España will enable the genetic identification of retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA) in affected patients.

17 Jul 2023

FDA-approved drug keeps light-sensitive photoreceptors alive in lab models of LCA 10

A National Institutes of Health team has identified a compound already approved by the U.S. Food and Drug Administration that keeps light-sensitive photoreceptors alive in three models of Leber congenital amaurosis type 10 (LCA 10), an inherited retinal ciliopathy disease that often results in severe visual impairment or blindness in early childhood.

29 Mar 2023

CRISPR-based genome editing restores the vision of mice with retinitis pigmentosa

Researchers in China have successfully restored the vision of mice with retinitis pigmentosa, one of the major causes of blindness in humans.

17 Mar 2023

New gene therapy approach could help prevent blindness in children with Leber congenital amaurosis

Researchers from the National Eye Institute have developed a gene therapy that rescues cilia defects in retinal cells affected by a type of Leber congenital amaurosis (LCA), a disease that causes blindness in early childhood.

8 Sep 2022

Virus-like particles as delivery vehicles for gene-editing

Scientists deliver gene-editing proteins through the use of engineered virus-like particles, which mediated efficient base editing both in vitro and in vivo.

13 Jan 2022

Gene therapy for common forms of congenital blindness shows promise to improve vision

A new gene therapy for one of the most common forms of congenital blindness was safe and improved patients' vision, according to initial data from a clinical trial led by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.

19 Apr 2021

Single injection of RNA therapy improves vision in patient with genetic form of childhood blindness

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.

1 Apr 2021

Gene therapy restores vison in dogs with severe form of Leber congenital amaurosis

Gustavo Aguirre and William Beltran, veterinary ophthalmologists and vision scientists at the University of Pennsylvania School of Veterinary Medicine, have studied a wide range of different retinal blinding disorders.

31 Mar 2021

NEI scientists develop and test a promising gene therapy strategy for rare eye disease

Scientists at the National Eye Institute have developed a promising gene therapy strategy for a rare disease that causes severe vision loss in childhood.

28 Jan 2021

Large study provides new understanding of genome diversity in Ukrainian population

Today, the largest study of genetic diversity in Ukraine was published in the open science journal GigaScience.

13 Jan 2021

Study uncovers a promising therapeutic target for retinal dystrophies

A team of LSU Health New Orleans researchers reports for the first time that deleting one of the inhibitors of the RPE65 gene in a mouse model that carries a human disease mutation prevents degeneration of cone photoreceptors that are used for daytime high-resolution color vision.

1 Dec 2020

Newly developed light-sensing protein restores vision in blind mice

A newly developed light-sensing protein called the MCO1 opsin restores vision in blind mice when attached to retina bipolar cells using gene therapy.

22 Oct 2020

Study helps restore retinal and visual functions of mice models of inherited retinal disease

A breakthrough study, led by researchers from the University of California, Irvine, results in the restoration of retinal and visual functions of mice models suffering from inherited retinal disease.

20 Oct 2020

Preclinical data of Ocugen's OCU400 genetic modifier published in Nature Gene Therapy

Ocugen, Inc., a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat rare and underserved ophthalmic diseases, announced today the publication in Nature Gene Therapy of preclinical data of nuclear hormone receptor gene NR2E3 as a genetic modifier and therapeutic agent to treat multiple retinal degenerative diseases.

3 Mar 2020