Leber Congenital Amaurosis News and Research

RSS

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

Gene-editing technique CRISPR trialed for first time as cancer treatment

The go-ahead has been given for the first trials using CRISPR as an alternative therapy for treating cancer.

6 Nov 2019

Johns Hopkins researchers develop new way to deliver sight-saving gene therapy to the retina

In experiments with rats, pigs and monkeys, Johns Hopkins Medicine researchers have developed a way to deliver sight-saving gene therapy to the retina.

From Johns Hopkins Medicine 30 Sep 2019

7-year-old treated with FDA-approved gene therapy for Leber congenital amaurosis

When Zion Grant was 3 months old his mother Daisy Dodd noticed he wasn't tracking her with his eyes. Zion would fixate on a light in the room, but not on her face.

24 Sep 2019

New therapy for childhood blindness shows 'very promising' results

A new therapy aimed at improving the sight of people with one of the most common forms of childhood blindness, has shown 'very promising' initial results, according to a study involving UCL researchers.

18 Dec 2018

Intraocular injection improves vision in a form of congenital retinal blindness

A new treatment for patients with a form of congenital retinal blindness has shown success in improving vision, according to results published today in Nature Medicine led by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania.

18 Dec 2018

Gene therapy for rare genetic blindness wins prestigious Champalimaud Vision award

A team of seven researchers collaborating from United States and Britain have found a way to use gene therapy to cure a rare form of childhood blindness that occurs due to a genetic predisposition.

5 Sep 2018

Study finds genetic treatment for inherited retinal disorder

A new study presented at the 121st Annual Meeting of the American Academy of Ophthalmology (AAO) 2017, for the first time found a novel gene therapy that can improve the eyesight of patients with inherited retinal disease.

13 Nov 2017

New gene therapy for blindness may soon be reality

Patients who had lost their sight to an inherited retinal disease could see well enough to navigate a maze after being treated with a new gene therapy, according to research presented today at AAO 2017, the 121st Annual Meeting of the American Academy of Ophthalmology.

10 Nov 2017

FDA advisory committee vote 16-0 for first even gene therapy for rare blindness

A panel of advisors to the United States Food and Drug Administration (FDA) has voted unanimously in favor of an experimental gene therapy to treat patients with a rare kind of hereditary blindness.

16 Oct 2017

Suppression of thyroid hormone receptor protects cone cells in mouse models of retinal diseases

New research published online in The FASEB Journal suggests that the suppression of thyroid hormone receptor activity locally in the retina protects cone photoreceptor cells in mouse models of human retinal degenerative diseases

11 May 2017

OIST scientists use zebrafish model to unravel causes behind human LCA

Newborns babies can be at risk of congenital blindness, presenting sight defects due to lesions or to genetic mutations in their genome.

5 Apr 2017

Penn scientists find striking similarities between human patients with LCA and dogs

Ciliopathies are diseases that affect the cilia, sensory organelles that most mammalian cells possess and which play a critical role in many biological functions.

30 Aug 2016

Study shows children with rare eye disease have greatest benefit from gene therapy

Scientists at Oregon Health & Science University's Casey Eye Institute and Baylor College of Medicine's Cullen Eye Institute published findings from a two-year Phase I clinical trial in the journal Ophthalmology, which showed that children had the greatest benefit from gene therapy for treatment of Leber congenital amaurosis (LCA) or severe early childhood onset retinal degeneration (SECORD).

18 Apr 2016

Gene therapy restores visual function in mouse model of LCA1

Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months.

1 Sep 2015

Genetic finding could lead to new treatments for people living with blindness, vision loss

Finding genes for retinal degenerations has immediate benefits for people living with blindness and vision loss, their families, and their physicians. Establishing a genetic cause confirms the clinical diagnosis at the molecular level, helps predict the future visual prognosis, suggests therapies, and allows some patients to join clinical trials. While more than 200 genes for retinal degenerations have been identified, approximately 40-50% of cases remain a mystery.

13 Jan 2015