A new target for Rett syndrome may address the underlying pathology caused by the loss of MECP2.


Lotje De Witte

Lotje De Witte’s research uses novel human microglia models that could create new Rett syndrome treatment development opportunities.

Dr. Hilde Van Esch

Dr. Hilde Van Esch's work aims to recreate Rett syndrome regression for the first time in an animal model.

Dr. Thorsten Stafforst

Dr. Thorsten Stafforst is developing a new RNA Editing method to convert the mutated cells of patients with Rett syndrome into healthy ones more safely and easily than other gene modifying therapy approaches.


Dr. Nicoletta Landsberger
Crystal Zhao, PhD
Dr. Davut Pehlivan
Michaela Fagiolini, PhD
Nicholas Katsanis, PhD
Colleen Niswender, PhD and Rocco Gogiliotti, PhD
Daniela Brunner, PhD, Early Signal Foundation
Jean-Christophe Roux, PhD, Inserm UMR 1251, Marseille Medical School
Susan A. Rose, PhD, Albert Einstein College of Medicine
Kyle Fink, PhD, University of California- Davis
Colleen Niswender, PhD – Vanderbilt University
Dr. Jenny Downs, Telethon Kids Institute in Australia
John M. Bissonnette, MD of Oregon Health and Science University
Carla J. Shatz, PhD of Stanford University
Keerthi Krishnan, PhD of Cold Spring Harbor Laboratory
Omar Khwaja, MD, PhD, MCRP of Children’s Hospital Boston
Rajiv R. Ratan, MD, PhD of Winifred Masterson Burke Medical Research Institute, Weill Medical College of Cornell
Lisa Monteggia, PhD, University of Texas Southwestern Medical Center
Zhaolan “Joe” Zhou, PhD, University of Pennsylvania, School of Medicine
Monica J. Justice, PhD, Baylor College of Medicine
Yi Eve Sun, PhD, University of California, Los Angeles
Liang Zhang, MD, PhD, University Health Network, Toronto Western Research Institute
John Christodoulou, AM, MB, BS, PhD, FRACP, FFSc, FRCPA, CGHGSA, Children’s Hospital at Westmead, Sydney, Australia
Aleksandra Djukic, MD, PhD, Tri-State Rett Syndrome Center, Montefiore Medical Center, Albert Einstein College of Medicine
Steven Gray, PhD, University of North Carolina at Chapel Hill
Investigator Spotlight: Qiang Chang, PhD, University of Wisconsin-Madison
Jeffrey Neul, MD PhD, Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and Baylor College of Medicine
Huda Y. Zoghbi, MD, Howard Hughes Medical Institute, Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and Baylor College of Medicine
Gail Mandel, PhD, Howard Hughes Medical Institute, Vollum Institute, Oregon Health and Science University

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