Mark Shen, PhD

Dr. Shen is a translational neuroscientist and tenured Associate Professor at the University of North Carolina in Psychiatry, Neuroscience, and the Carolina Institute for Developmental Disabilities (CIDD), where he is the founding Director of the CIDD Clinical Trials Program. The goal of his lab is to identify early biological markers and therapeutic targets for neurodevelopmental disorders (NDDs), including autism, fragile X, Down syndrome, Rett syndrome, Dup15q, and Angelman syndrome. This research spans the translational pipeline, bridging clinical research, preclinical models, and clinical trials. We conduct longitudinal neuroimaging and deep phenotyping of infants to identify the earliest brain features and clinical symptoms of NDDs. We then collaborate with preclinical scientists to reverse-translate these pediatric findings into mechanistic studies using animal and cellular models. Since launching the CIDD Clinical Trials Program in 2018, he has built a team of physicians and clinical trialists to advance genetic medicine trials for children with NDDs. Dr. Shen serves as PI of two Phase 1/2a clinical trials delivering antisense oligonucleotides (ASOs) in children with Angelman syndrome to reactivate the silenced genetic allele. Phase 1 results were recently published in Nature Medicine. Prior to his research career, he worked for six years in the community as a behavioral therapist for children and adults with profound autism. He then completed his PhD with David Amaral at the UC Davis MIND Institute and his postdoctoral fellowship with Joe Piven at UNC. Dr. Shen serves on the Executive Committee, Genetics Core, and MRI Core of the Infant Brain Imaging Study (IBIS), an NIH Autism Center of Excellence multi-site network that has followed infants from age 6 months to 18 years.