Gene Therapy for Parkinson's

This bio.com newsfeature highlights a provocative genetic therapy for Parkinson's by scientists affiliated with Northwestern University.

By placing the RNA interference into a crippled, non-disease-causing virus, scientists in the Bohn lab have been able to deliver the RNA interference tool to the brain of rats and turn off the alpha-synuclein protein in neurons. "This is the first step in developing a new therapy for Parkinson's disease based on molecular knowledge of the disease," said Mohan K. Sapru, research assistant professor of pediatrics, who is first author on the study and co-inventor of the gene therapy technology.

"It may also be useful for other diseases of the brain, such as dementia with Lewy bodies, a disease also characterized by Lewy bodies in the brain," Sapru said.

The Bohn lab will subsequently test this gene therapy in mouse models of the disease. If the RNA interference approach works in the mouse, a gene therapy based on silencing the _alpha-synuclein gene will be developed for clinical trials for Parkinson's patients.

This technique apparently uses a viral vector to deliver a RNAi therapy to neurons to prevent a troublesome protein from being produced. Noncoding RNAs, including RNAi, represent a vast new continent ripe for exploration, survey, subdivision, and finally one the productive settlers (scientists) move in, tremendous progress at a dizzying rate.