SAN DIEGO -- Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders. In new research presented today, scientists successfully used gene therapy to slow the progression and improve symptoms of disorders such as amyotrophic lateral sclerosis and Parkinson's disease. The findings were presented at Neuroscience 2018, the annual meeting of the Society for Neuroscience and the world's largest source of emerging news about brain science and health.
Gene therapy typically employs an inactivated virus to carry new genetic cargo into cells, altering specific genes to treat or prevent a disease. Researchers might replace a mutated gene with a healthy copy of the gene, turn off a disease-causing gene, or add a new gene to the body to help fight a disease.
Although gene therapy is a promising treatment option for a limited number of conditions, including certain cancers, the technique is still experimental for most diseases, with ongoing research to ensure they will be safe and effective in human patients. Animal studies are a key part of the process by which an experimental gene therapy treatment goes to clinical trial.
Today's new findings show that:
"Gene therapy holds the promise to transform the lives of patients with incurable neurological diseases," said press conference moderator Fredric Manfredsson, PhD, of Michigan State University. "The research presented today represents important and exciting steps toward being able to prevent and treat disorders that currently have no cure, such as Parkinson's disease and Alzheimer's disease."
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This research was supported by national funding agencies including the National Institutes of Health, as well as other public, private, and philanthropic organizations worldwide. Find out more about gene therapy on BrainFacts.org .
Related Neuroscience 2018 Presentation Special Lecture: The Genetics, Neurobiology, and Evolution of Natural Behavior Tuesday, Nov. 6, 10-11 a.m., SDCC Ballroom 20