Gene Therapy
Vocal fold paralysis therapies
A collaboration between PNR&D surgeons and scientists from the Otolaryngology-Head and Neck Surgery Department and Department of Neurology focuses upon the problem of paralyzed vocal cords. A paralyzed vocal cord can cause severe voice and swallowing problems, and if both vocal cords are paralyzed, breathing trouble usually develops. Current treatment options are unable to restore proper vocal cord movements. We believe that a novel treatment approach using virally-mediated gene therapy may hold the answer to this serious clinical problem. A laboratory model of vocal cord paralysis has been developed, and experiments using viral vectors containing candidate therapeutic genes are underway.
Gene therapy
One hurdle to the treatment of motor neuron diseases like ALS is the inability to effectively target therapies to affected cells in the spinal cord. To overcome this hurdle, PNR&D investigators are currently developing and optimizing gene therapy approaches to deliver neuroprotective factors to the nervous system. Using genetically engineered viruses, genes for neural growth factors can be retrogradely transported through motor neuron axons following intramuscular injection to the motor neuron cell bodies in the spinal cord. One such growth factor, insulin-like growth factor-I (IGF-I), elicits neuroprotective properties in both cellular and animal models of ALS and can be targeted to MNs using this approach. Similarly, vectors delivering a transcription factor designed to upregulate the neuroprotective growth factor vascular endothelial growth factor (VEGF) in MNs have also been successfully developed and are currently being tested in various disease and injury models. Results of ongoing gene therapy techniques in the PNR&D are anticipated to provide a novel means to administer and target therapies to the spinal cord following intramuscular injection.
