Genetic Modifiers: Lessons from Nervous System Disorders

Tuesday, September 21, 2021
Genetic Modifiers graphic/flyer

SAVE THE DATES: September 21, 22 & 23 | 10 a.m. - 2 p.m.
Virtual Event (Zoom)

This workshop will bring together a diverse group of researchers with expertise in genetics, genomics, neurology, pathophysiology, and preclinical translation. Understanding genetic factors that modify the onset, progression or severity of diseases can lead to novel therapeutics, facilitate clinical trial design, and enable personalized medicine. The purpose of this workshop is to promote collaborations, confront challenges and highlight successful strategies for genetic modifier discovery, validation, and clinical implementation as part of the NIH Blueprint mission to foster transformative discoveries in neuroscience.


  • Day 1: Modifier discovery in human cohorts and model systems, resources available and successful analytical approaches
  • Day 2: Validation and characterization of modifiers, determining mechanisms of disease modification and mapping modifier pathways
  • Day 3: Clinical applications of modifiers as therapeutic targets, in design of clinical trials and in precision medicine

Who Can Attend:  Attendance will be free and open to all interested researchers, patients, advocacy groups


NIH workshop, co-chaired by:
Susan Ackerman, Ph.D. (University of California, San Diego)
Monica Justice, Ph.D. (The Hospital for Sick Children, University of Toronto)
Trudy MacKay, Ph.D. (Clemson University)
Huda Zoghbi, M.D. (Baylor College of Medicine)
Stephan Zuchner, M.D., Ph.D. (University of Miami)
Marcy E. MacDonald, Ph.D. (MGH/Harvard )

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Form Approved OMB# 0925-0648 Exp. Date 06/2024