A new $418,000 grant from the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, will allow Anton Reiner, PhD, and Tayebeh Pourmotabbed, PhD, of the University of Tennessee Health Science Center (UTHSC) to research a possible new gene therapy treatment approach for Huntington’s disease.
Huntington’s disease is a hereditary degenerative brain disease that ultimately results in the destruction of the primary thinking and planning part of the brain called the cerebral cortex, and the destruction of a major motor control region of the brain known as the basal ganglia.
Drs. Reiner and Pourmotabbed’s research will be conducted through a grant titled, “Development of DNAzyme Gene Therapy for Huntington’s Disease.” They will both serve as principal investigators on this award. The funds will be distributed over two years.
Dr. Reiner is a professor in the Department of Anatomy and Neurobiology in the UTHSC College of Medicine, and Dr. Pourmotabbed is a professor in the Department of Microbiology, Immunology and Biochemistry in the College of Medicine.
The researchers will develop and test a novel gene therapy, which will be evaluated for its effectiveness in mice that have been genetically engineered to have the disease.
The gene therapy employs molecules called DNAzymes that have been designed to selectively prevent the formation of the mutant protein huntingtin, which causes Huntington’s disease.
“These molecules have the advantage compared to other forms of gene therapy that they are stable and could, in humans, be injected intravenously,” said Dr. Pourmotabbed. “By contrast, other gene therapies for the disease require injection into the brain or the lumbar vertebral canal. In our initial studies in mice with Huntington’s disease, we found that intraperitoneal injections greatly reduced mutant protein in the brain and peripheral organs, and reduced the motor symptoms of the disease,” said Dr. Reiner.
The immediate goal of this study is to determine how long this benefit lasts, how much DNAzyme must be administered to achieve it, and whether there are any toxic side effects.
The National Institute of Neurological Disorders and Stroke is dedicated to research and the dissemination of information on the brain and nervous system in efforts to reduce neurological disease. For more information, visit www.ninds.nih.gov.
The National Institutes of Health (NIH), the nation’s medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit www.nih.gov.