N2 - Neuroscience in the Clinic: Antisense Oligonucleotide (ASO) Therapy

Event Time: Monday April 23, 2018 3:30 pm to 5:30 pm
Topic(s): Neuromuscular and Clinical Neurophysiology (EMG)
Director(s): John Day MD, PhD, Richard Finkel MD
Description: Ongoing clinical research has already identified the genetic cause of hundreds of distinct disorders, most notably in the area of neuromuscular neurology. While knowing the underlying genetic cause provides a clear target for course-modifying treatments, the ability to successfully and safely modify genes in the clinical world has remained difficult until very recently. After decades of development, technologies are now being successfully used to modify pathogenic mutations using antisense technologies. The faculty will discuss the range of antisense technologies now in clinical investigation, and the multiple methods by which they can alter expression of disease-causing mutations, focusing on current successes such as for Spinal Muscular Atrophy, and future targets. Specific cases will be discussed to show how antisense technologies are being used clinically and how they will impact current and future care of neuromuscular disorders.
Completion Message: Participants will be able to identify different antisense oligonucleotide chemistries and their relative values. They will understand the biodistribution and pharmacokinetics of some antisense oligonucleotides, and the various ways in which antisense oligonucleotides can impact normal and pathogenic gene expression. They will get an update on recently approved antisense treatments that are now commercially available, as well as those that are currently in development.
CME Credits: 2
Core Competencies: Patient Care, Medical Knowledge

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