The 2019 IAMSE meeting offers many opportunities for faculty development and
Getting Translational Research Into the Classroom: The Promise of Gene Silencing Therapy for Diseases of the Aging Nervous System
Presenter: Don Cleveland
Plenary Address: Sunday, June 9, 8:45 AM – 9:45 AM
Remarkable advances are altering therapy options for previously intractable diseases, including for diseases of the aging nervous system. With an aging population and therapies on the horizon to alter the course of neurogenerative diseases, today’s medical curriculum should include the rationale and promise of these emerging approaches. Recent advances in gene therapy research have highlighted how sustained gene silencing within neurons and non-neurons throughout the nervous system has been achieved using a clinically feasible approach with “designer DNA drugs”. This approach has slowed disease progression in genetic mimics of inherited ALS and has produced prolonged, partial disease reversal in models of Huntington’s disease. A similar designer DNA drug has successfully corrected an error in the assembly of an RNA intermediate, thereby restoring production of the gene product whose absence is the cause of spinal muscular atrophy, one of the most abundant inherited diseases of children. Furthermore. for the most frequent genetic cause of both ALS and the second most frequent dementia (frontal temporal dementia), single dose administration of a designer DNA drug has reduced the synthesis of the toxic product of the mutated gene and prevented age-dependent cognitive disease. Building on these advances in clinical science, this talk will highlight the importance of cutting edge basic science for medical science students in training, highlight approaches for its successful integration, and explore how scientists and educators can work together to deliver this important aspect of healthcare into traditional anatomy and physiology courses.
For more information on Dr. Cleveland’s plenary address, and to register for the 23rd Annual IAMSE Meeting, please visit www.IAMSEmeeting.org.