ANA Highlights Gene Therapy



The ANA Highlights Gene Therapy program is chaired by 2024 ANA President, Dr. M. Elizabeth Ross, Nathan Cummings Professor and Head, Laboratory of Neurogenetics and Development, Director, Center for Neurogenetics at Weill Cornell Medicine and Dr. Bryan Traynor, Senior Investigator at the National Institute on Aging (NIA) and the National Center for Advancing Translational Sciences (NCATS), where he leads the RNA Therapeutics Laboratory. This course is brought to you by the ANA Education Innovation Committee, currently chaired by Dr. Michelle C. Johansen, Assistant Professor of Neurology, Cerebrovascular Division at the Johns Hopkins Hospital. The ANA received a grant from Sanofi in support of this activity.

Advances in research have yielded different mechanisms for modifying genes that cause disease. This course includes four videos, the first three outline the following mechanisms and the course closes with a moderated panel discussion about the applications and current state of the research in this field.

  • Adeno-Associated Virus with Stephen G. Kaler, MD, Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, OH
  • Anti-Sense Oligonucleotides with Harry T. Orr, PhD, Director of Institute for Translational Neuroscience, University of Minnesota (
  • CRISPR with Claire Clelland, PhD, MD, MPhil, Assistant Professor of Neurology, UCSF Weill Institute for Neurosciences & UCSF Memory & Aging Center (
Interviewer/Producer: Dr. Bryan Traynor
Interviewer/Producer: Dr. M. Elizabeth Ross