University of Florida Movement Disorders Center

Gene therapy for Parkinson's disease moves forward in animals

Excerpts from a UF Health Science Center article by John Pastor (full article here)

An international team of scientists has used gene therapy in two separate studies to renew brain cells and restore normal movements in monkeys and rats with a drug-induced form of Parkinson's disease.

Ron Mandel, Ph.D., (left) a scientist with the University of Florida's McKnight Brain Institute and Genetics Institute, was part of the research team that found neuroprotective benefit from treatment in monkeys. In tests with 31 monkeys, including a control group, scientists inserted copies of a gene to produce GDNF into a region in the front part of the brain called the striatum. They then induced Parkinson-like conditions by introducing a drug to destroy the dopamine-producing cells. Seventeen weeks after that, not only did the GDNF-treated monkeys show improvement in performing tasks, analysis of brain tissue showed the animals' dopamine systems were actually spared by the treatment.

In efforts to reverse impaired movements in rats, scientists used 33 animals with severe dopamine depletion and transferred a gene to provide a source of L-dopa production into the animals' striata. Before receiving the treatment, all animals had limited use in their left paws. After treatment, the animals receiving the therapeutic enzyme mixture show complete recovery in their paws. Researchers say not only did the rats recover substantial degrees of function in their impaired forelimbs, continuous levels of L-dopa were being produced in their brains, blocking side effects.

Read the full article
Read an article in the Gainesville Sun about the research
Read more about Gene Therapy research for Parkinson's


Home   |   Medicine   |   Surgery   |   Education   |   Research   |   Team   |   Conatct Us