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Stem Cells and the Future of Autism Research and Treatment

Induced pluripotent stem cells (iPSCs) have the extraordinary property of being able to differentiate into any somatic cell type, including cells of the neural lineage. This has rendered possible the preparation of ‘diseases in a petri dish’ wherein iPSCs from individuals with specific neurological conditions can be cultured and studied with electrophysiological and molecular biological techniques. These assays can be combined with combinatorial chemical libraries, as well as cDNA and RNAi libraries, to screen for agents that target disease-relevant pathways, thus opening new windows for therapeutic discovery.

Ultimately, stem cells could be used to repair cellular assemblies in regions of the brain thought to be affected in autism, such as Purkinje cells in the cerebellum. In view of the surprising genetic and phenotypic heterogeneity in autism-related disorders, the ability to carry out these assays in a patient-specific genetic background will remove one serious confound in the search for mechanisms and remediation.

Matthew Anderson, MD, Ph.D., Beth Israel Deaconess Medical Center

Michael E. Coulter

Daniel Ebert, M.D., Massachusetts General Hospital

Modeling Pathogenesis & Treatment of Autism Spectrum Disorders Using Patient-Specific Stem Cells: Fragile X Syndrome
Stephen J. Haggarty, Ph.D., Massachusetts General Hospital

Dissecting the Mechanisms of Cellular Reprogramming
Konrad Hochedlinger, Ph.D., Massachusetts General Hospital

Ray Kelleher, M.D., Ph.D., Massachusetts General Hospital

Mustafa Sahin, MD, Ph.D., Children’s Hospital Boston

An Autism Stem Cell Biorepository: Gaining Insights from Induced Pluripotent Stem Cell Technology
Philip H. Schwartz, Ph.D., Children’s Hospital of Orange County

Induced Conditional Self-renewing Progenitor (ICSP) Cells
Richard L. Sidman, M.D., Beth Israel Deaconess Medical Center

Timothy Yu, MD, Ph.D., Lurie Family Autism Center, Massachusetts General Hospital

Andrew W. Zimmerman, MD, Lurie Family Autism Center, Massachusetts General Hospital 


The Nancy Lurie Marks Family Foundation, Wellesley, MA