Leber Congenital Amaurosis News and Research

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Researchers identify genes that hinder CRISPR editing efficiency

Like a delivery driver navigating crowded city streets, a gene-therapy-toting lipid nanoparticle faces a gauntlet of potential detours on its journey toward a cell's nucleus.

13 Aug 2026

Retinal organoids provide a powerful tool to diagnose and study Leber congenital amaurosis

Leber Congenital Amaurosis (LCA) is an inherited retinal disease leading to severe vision impairment from early infancy, affecting 2-3 out of every 100,000 newborns. LCA is caused by variants in certain genes from which proteins essential for retinal cell function and survival are made.

3 Dec 2025

Breakthrough research reveals gut-eye connection in vision loss

Inherited retinal degeneration (IRD) encompasses various disorders characterized by progressive loss of retinal photoreceptor cells, ultimately leading to vision loss and blindness.

2 Apr 2025

New genetic medicine offers life-changing vision improvements for blind children

Four young children have gained life-changing improvements in sight following treatment with a pioneering new genetic medicine through UCL Institute of Ophthalmology and Moorfields Eye Hospital, with the support of MeiraGTx.

22 Feb 2025

Why predictive retina models could be the future of IND applications

Discover how retina models are revolutionizing rare disease treatment and accelerating drug development.

From Newcells Biotech 22 Nov 2024

Gene therapy shows dramatic vision improvement for rare inherited condition

The vision of people with a rare inherited condition that causes them to lose much of their sight early in childhood was 100 times better after they received gene therapy to address the genetic mutation causing it.

5 Sep 2024

CRISPR-based gene editing shows promise in treating rare form of blindness

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.

6 May 2024

CRISPR gene editing shows promise for treating individuals with a form of inherited blindness

Results from a groundbreaking clinical trial of CRISPR gene editing in 14 individuals with a form of inherited blindness show that the treatment is safe and led to measurable improvements in 11 of the participants treated.

6 May 2024

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

Leber Congenital Amaurosis News and Research

Researchers identify genes that hinder CRISPR editing efficiency

Retinal organoids provide a powerful tool to diagnose and study Leber congenital amaurosis

Breakthrough research reveals gut-eye connection in vision loss

New genetic medicine offers life-changing vision improvements for blind children

Why predictive retina models could be the future of IND applications

Gene therapy shows dramatic vision improvement for rare inherited condition

CRISPR-based gene editing shows promise in treating rare form of blindness

CRISPR gene editing shows promise for treating individuals with a form of inherited blindness

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

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

Can genome editing transform ocular disease treatment?

New agreement to facilitate genetic diagnosis of hereditary retinal dystrophies

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

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

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

Virus-like particles as delivery vehicles for gene-editing

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

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

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

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

Industry Focus eBook - Cell & Gene Therapy (1st Edition) eBook

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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