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Wednesday, November 02, 2005

Treatments for LCA Tested in Mice 

Two approaches to treating a form of Leber's Congenital Amaurosis (LCA) caused by the loss of an enzyme known as LRAT have been tested together by researchers at Case Western Reserve University in the United States. LCA is a form of retinal degeneration that results in congenital blindness. The researchers conducted their experiments, which combined gene therapy and a drug treatment, in mice.


The team hopes that if the treatments are used together, treatment with oral retinoids could begin in infancy to avoid early sight loss and the difficulties associated with surgery in very young patients. And when patients are older, long-lasting drug-free treatment could be done by surgically introducing gene therapy. This study marks the first step in finding out whether these treatments will work effectively and safely in humans.
'Successful tests of new treatments in mice for eye disease causing irreversible blindness in humans' (Case Western Reserve University media release at EurekAlert, 1 November 2005).


The research is reported in this month's issue of the peer-reviewed, open access journal PLoS Medicine. Information is also available in 'Treatment for Leber Congenital Amaurosis' (News-Medical.Net, 1 November 2005).

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