Gene Therapy Helps Children Suffering From Batten Disease

Washington (dbTechno) – According to a team of researchers from the New York Presbyterian Hospital / Weill Cornell Medical Center, gene therapy is safe and effective to help children suffering from the rare brain disorder, Batten disease.

Batten disease is a very rare, genetic brain disorder which grows over time, and usually fatal in children before they become teenagers.

It is also known as Late Infantile Neuronal Ceroid Liopfuscinosis (LINCL).

Researchers have reported on Tuesday that their experimental treatment is safe and effective to treat children with this rare disorder.

The treatment has the virus carrying a corrective gene, which was placed into the brain.

Researchers have stated that this gene therapy slowed the progression of the disease in 80% of the children they tested for the study.

Though this is not a cure, it is a huge step in the right direction to help with the treatment of LINCL.

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

• Share
• Tweet
• "&title=""">