CHICAGO -- Promising findings from preclinical animal studies and postmortem human brain data show the potential of gene therapy for treating currently incurable neurological disorders. The findings were presented at Neuroscience 2019, the annual meeting of the Society for Neuroscience and the world's largest source of emerging news about brain science and health.
Gene therapy is an experimental technique that involves altering specific genes to treat or prevent disease. Typically, an inactivated virus is used to carry the genetic cargo into cells. 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. Gene therapy is a promising treatment for a number of currently incurable neurological diseases, such as Alzheimer's disease, amyotrophic lateral sclerosis, and Parkinson's disease.
Today's new findings show that:
"Gene therapy holds the promise to transform the lives of patients with incurable neurological diseases," said Jeff Kordower, PhD, a professor at Rush University who studies aging and neurodegenerative disease. "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."
This research was supported by national funding agencies including the National Institutes of Health and private funding organizations. Find out more about gene therapy on BrainFacts.org .
Related Neuroscience 2019 Presentation
Roundtable: Gene Therapy in Neurological Diseases
Wednesday, Oct. 23, 8:30 - 11:00 a.m., Room N230B
Gene Therapy Press Conference Summary
A Gene Therapy Approach to Directly Reprogram Reactive Astrocytes Into Functional Motor Neurons in SOD1G93A ALS Mouse Model
Gong Chen, guc2@psu.edu , Abstract 475.10
Neuronal Expression of Nusc1, a Single-Chain Variable Fragment Antibody Against Abeta Oligomers, Protects Synapses and Rescues Memory in Alzheimer's Disease Models
Sérgio Ferreira, ferreira@bioqmed.ufrj.br , Abstract 447.13
Investigating Enhanced Gene Transfer to the Mouse Central Nervous System Using Modified Viral Vectors
Sophie. N. Mathieson, matso768@student.otago.ac.nz , Abstract 175.13
Long-term Post-Mortem Studies Following Neurturin Gene Therapy in Advanced Parkinson's Disease
Jeff Kordower, Jeffrey_Kordower@rush.edu , Abstract 383.05
Scalable cGMP Compliant Expansion of Human Fetal and iPSC Derived Neural Progenitor Cells
Clive Svendsen, Clive.Svendsen@cshs.org , Abstract 475.13
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About the Society for Neuroscience
The Society for Neuroscience is the world's largest organization of scientists and physicians devoted to understanding the brain and nervous system. The nonprofit organization, founded in 1969, now has nearly 37,000 members in more than 90 countries and over 130 chapters worldwide.