New therapeutic targets for treating Parkinson’s disease

Scientists at the National Institutes of Health have used RNA interference (RNAi) technology to reveal dozens of genes which may represent new therapeutic targets for treating Parkinson’s disease. The findings also may be relevant to several diseases caused by damage to mitochondria, the biological power plants found in cells throughout the body.

“We discovered a network of genes that may regulate the disposal of dysfunctional mitochondria, opening the door to new drug targets for Parkinson’s disease and other disorders,” said Richard Youle, Ph.D., an investigator at the National Institute of Neurological Disorders and Stroke (NINDS) and a leader of the study.

Dr. Youle collaborated with researchers from the National Center for Advancing Translational Sciences (NCATS).

Dr. Youle and his colleagues worked with Scott Martin, Ph.D., a coauthor of the paper and an NCATS researcher who is in charge of NIH’s RNAi facility. The RNAi group used robotics to introduce small interfering RNAs (siRNAs) into human cells to individually turn off nearly 22,000 genes. They then used automated microscopy to examine how silencing each gene affected the ability of parkin to tag mitochondria.

“One of NCATS’ goals is to develop, leverage and improve innovative technologies, such as RNAi screening, which is used in collaborations across NIH to increase our knowledge of gene function in the context of human disease,” said Dr. Martin.

“This study shows how the latest high-throughput genetic technologies can rapidly reveal insights into fundamental disease mechanisms,” said Story Landis, Ph.D., director of the NINDS. “We hope the results will help scientists around the world find new treatments for these devastating disorders.”

Source: National Institute of Neurological Disorders and Stroke, USA

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