Stem Cell Based Technologies in Autism Research

A significant limitation on the investigation of neurological and psychiatry diseases including autism is the inability to study disease processes in affected human cells, namely neurons. Ideally, drugs and therapeutics for brain disorders should be tested on relevant human neurons before being used on participants in clinical trials. While the direct use of tissues from human disease populations is scientifically desirable, obtaining them is rarely safe or feasible. Recently, it has become possible to reprogram cells derived from a skin biopsy or blood sample of an individual into induced pluripotent stem cells, or iPSCs. Additionally, significant progress has been made to differentiate iPSC into defined neuron sub-types. Many laboratories are now able to reproducibly reprogram human cells into induced pluripotent stem cell (iPSC) lines, engineer the correction of patient mutations and differentiate resulting cell lines into patient derived models of disease in a dish. As a result, many investigators are starting to develop meaningful phenotypic assays for drug screening in support of syndromic or non-syndromic forms of autism. This panel will discuss the recent advances, opportunities and challenges in this burgeoning field of research.
Thursday, May 2, 2019: 10:30 AM-12:30 PM
Room: 524 (Palais des congres de Montreal)
Panel Chair:
M. Sahin
10:55 AM
Complete Disruption of Autism-Susceptibility Genes By Gene-Editing Predominantly Reduces Functional Connectivity of Isogenic Human Neurons
E. Deneault S. H. White D. C. Rodrigues J. Ross M. Faheem K. Zaslavsky Z. Wang R. Alexandrova G. Pellecchia W. Wei A. Piekna G. Kaur J. Howe V. Kwan B. Thiruvahindrapuram S. Walker A. C. Lionel P. Pasceri D. Merico R. K. Yuen K. K. Singh J. Ellis S. W. Scherer