Pierson Lab
The Pierson Laboratory is devoted to understanding the pathological mechanisms involved in rare and undiagnosed pediatric neurogenetic disorders. Tyler Mark Pierson, MD, PhD, has extensive experience in the phenotyping and diagnosing of rare neurogenetic disorders using modern genomic technologies. Pierson did a clinical fellowship in the National Institute of Neurological Disorders and Stroke Neurogenetics Branch at the National Institutes of Health (NIH) and was a member of the Undiagnosed Diseases Program at the NIH. In addition to directing his laboratory, Pierson is also the director of the Pediatric Neurogenetics and Neuromuscular Clinic of Cedars-Sinai and the pediatric lead of the Cedars-Sinai Center for the Undiagnosed Patient.
The research goal of the Pierson Laboratory is to extend these diagnostic elements by using patient-derived induced pluripotent stem cells to understand the disease mechanisms associated with these rare pediatric neurogenetic disorders and to explore stem cell-based therapies for the delivery of therapeutic drugs, small molecules and proteins. The Pierson Lab is also involved in confirming neurogenetic diagnoses in known, but rare, neurogenetic disorders, as well as developing research to link rare presentations of neurogenetic disorders with new genes and causes.
The Pierson Laboratory is affiliated with the Cedars-Sinai Center for the Undiagnosed Patient, Board of Governors Regenerative Medicine Institute, Neurology & Neurosurgery Department, the Pediatric Neurogenetics Clinic and Pediatrics Department.

Personal Statement
I am a physician-scientist with more than 20 years of clinical and basic science research experience focusing on rare neurogenetic disorders. My goals are to help families with these disorders in the clinic as well as studying the disorders in my laboratory. I did my research training in the laboratory of Bert O’Malley, MD, working with the mifepristone-inducible gene system for gene therapy. I then completed pediatric neurology training at the Children’s Hospital of Philadelphia, followed by a fellowship in neurogenetics at the National Institutes of Health, as well as being an early member of the Undiagnosed Diseases Program at NIH prior to moving to Cedars-Sinai. At Cedars-Sinai, I have worked to organize the Pediatric Neurogenetics Clinic and am part of the Center for the Undiagnosed Patient. My laboratory research focuses on developing methods to better understand and treat neurodevelopmental and neurodegenerative disorders of childhood."
Tyler Mark Pierson, MD, PhD
Rare neurogenetic disorders are often caused by mutations that alter the function of important genes involved in neurodevelopment, or genes that can lead to neurodegeneration. Many of the genes involved in neurodevelopmental disorders are crucial to the choreography of brain development leading to issues with cognition and behavior. Alternatively, pediatric neurodegenerative disorders occur after normal brain development with subsequent dysfunction of cells within the nervous system leading to their demise. Our group studies both types of these disorders with patient-derived IPSCs, inducible-neurons and cerebral organoids to provide insight into neurologic function, as well as researches therapeutic interventions for affected families.
Collaboration
The Pierson Laboratory is supported by the Diana and Steve Marienhoff Fashion Industries Guild Endowed Fellowship in Pediatric Neuromuscular Diseases and the Fashion Industries Guild Endowed Fellowship in Undiagnosed Diseases.
External
- Undiagnosed Diseases Network, National Institutes of Health and UCLA
- Taroni Laboratory, Fondazione I.R.C.C.S. Istituto Neurologico "C. Besta" Milan, Italy
- Bert O’Malley Laboratory and Sophia Tsai Laboratory, Baylor College of Medicine, Houston, Texas
- Weimer Laboratory, Sanford Research, Sioux Falls, South Dakota
- Traynelis Laboratory and Yuan Laboratory, Emory University, Atlanta, Georgia
- Ward Laboratory, NIH/NINDS/Neurogenetics Branch, Bethesda, Maryland
- Dowling Laboratory, Hospital for Sick Children, Toronto, Ontario
- Young Laboratory and Walz Laboratory, University of Miami, Coral Gables, Florida
- Lachlan Jolly, PhD
- Michael Ibba, PhD
Meet Our Team
Learn more about the scientists, faculty members, investigators and other healthcare professionals of the Pierson Laboratory, whose dedicated efforts lead to groundbreaking discoveries.
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Biol Psychiatry. 2019 Jan 20;87(2): 100-112.
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