Gene Therapy Helps Children Fight Rare Brain Disease

May 14, 2008

A team of researchers from the New York Presbyterian Hospital / Weill Cornell Medical Center has found that gene therapy is an effective way to help chidlren suffering from a rare brain disease.New York (ChattahBox) – A team of researchers from the New York Presbyterian Hospital / Weill Cornell Medical Center has found that gene therapy is an effective way to help children suffering from a rare brain disease.

The rare genetic brain disorder is called Batten Disease, also known as Late Infantile Neuronal Ceroid Liopfuscinosis (LINCL).  This disease is usuall fatal in children before the age of 13.

Researchers tested an experimental gene therapy treatment and found it to be effective in treating the disease. It works by carrying a corrective gene into the brain.

Out of all of the children tested, the gene therapy slowed the progression of the disease 80% of the time.

The study has been published in the journal Human Gene Therapy.


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