Experimental Therapeutics - Gene Therapy for Childhood Neurological Disorders

Course Description
Participants will gain an understanding of the rapidly evolving landscape of gene therapy for neurological disorders of childhood. We will discuss the basic principles of gene vector development and delivery, and describe specific diseases for which gene therapy is currently approved for clinical use or available as an experimental treatment in clinical trials. Finally, we will discuss challenges facing gene therapy development, including cost and worldwide accessibility.

Learning Objectives

1. Understand the principles of gene therapy development and delivery.
2. Be able to describe how different gene therapy strategies can be used to treat specific neurologic diseases in children.

Impact Statements

1. Recognize genetic disorders that are amenable to treatment with gene therapy.
2. Identify opportunities for patients with rare diseases to participate in clinical trials of experimental therapies.

Organizer:

Toni Pearson, MBBS
Washington University School of Medicine, St. Louis, Missouri, USA

Gene Therapy Primer
Barry Byrne, MD, PhD
Child Health Research Institute, University of Florida, Gainesville, Florida, USA

Experiences with Gene Therapy for Childhood Neuromuscular Disorders
Diana Bharucha-Goebel, MD
Children’s National Hospital & National Institutes of Health, Washington, DC, USA

Targeting the Central Nervous System: Experiences with Gene Therapy for AADC Deficiency
Toni Pearson, MBBS

Panel Discussion – Present and Future Challenges in the Development of Universally Accessible New Therapies for Ultra-Rare Diseases
Carsten G. Bönnemann, MD
National Institute of Neurological Disorders and Stroke, Bethesda, Maryland, USA