Leber Congenital Amaurosis News and Research

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Study shows gene therapy can restore vision to children with Leber congenital amaurosis

Results from a breakthrough study, conducted by researchers at The Children’s Hospital of Philadelphia (CHOP) and the University of Pennsylvania School of Medicine, show that gene therapy has restored significant vision in five children and seven adults who were previously blind.

27 Oct 2009

Remarkable vision improvements after gene therapy for inherited blindness

One year after a trio of young adults received gene therapy for an inherited form of blindness, researchers have documented that the patients are still experiencing the same level of remarkable vision improvements previously measured within weeks.

13 Aug 2009

Discovery of new gene that causes Leber congenital amaurosis and retinitis pigmentosa

The team of Dr. Robert Koenekoop which includes Dr. Irma Lopez from the Research Institute of the MUHC at the Montreal Children's Hospital played a crucial role in the international collaboration that led to the discovery of a new gene that causes Leber congenital amaurosis (LCA) and retinitis pigmentosa (RP), two devastating forms of childhood blindness.

5 Mar 2009

Gene therapy restores vision in patients with rare congenital blindness

In a clinical trial at The Children's Hospital of Philadelphia, researchers from The University of Pennsylvania have used gene therapy to safely restore vision in three young adults with a rare form of congenital blindness.

27 Apr 2008

Gene-transfer technique trial for childhood blindness under way

Three decades have passed since gene therapy pioneer William W. Hauswirth, Ph.D., and his colleagues at the University of Florida began work on a virus that could safely deliver corrective genes into living animals.

19 Nov 2007

New understanding of Leber congenital amaurosis

People suffering from a severe retinal disease will sooner or later lose their eyesight considerably or even become completely blind.

7 Aug 2007

New genetic cause for Joubert syndrome

An international study by researchers at Seattle Children?s Hospital Research Institute, the University of Washington School of Medicine, and Radboud University in Nijmegen, Netherlands has identified a new genetic cause for Joubert syndrome (JS).

15 Jun 2007

Gene responsible for Leber Congenital Amaurosis

A MUHC-led study identifies a gene responsible for Leber Congenital Amaurosis (LCA), the most common cause of congenital blindness in infants and small children.

5 Jun 2007

First steps taken to treat human hereditary blindness

A team led by Krzysztof Palczewski, Ph.D., chair of pharmacology at the Case Western Reserve University School of Medicine, has taken the first steps in treating an eye disease causing irreversible congenital blindness in millions of people worldwide by successfully testing two new treatments in mice.

3 Nov 2005

Treatment for Leber Congenital Amaurosis

U.S. researchers have taken the first steps in treating an eye disease that causes irreversible blindness in humans by successfully testing two novel treatments in mice.

1 Nov 2005

Findings could lead to gene therapy cure for childhood blindness

In findings that could lead to curing some forms of congenital blindness through gene therapy, researchers at UCLA have discovered that RPE65, a gene missing in infants born with the blinding disease Leber congenital amaurosis, is also a key enzyme in the visual cycle. The identity of this enzyme has long been a mystery to scientists.

11 Aug 2005