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Mining the Mind: CRISPR-based genome-wide screens in iPSC-derived brain-disease models

This webinar is brought to you by the Science/AAAS Custom Publishing Office

Mining the Mind: CRISPR-based genome-wide screens in iPSC-derived brain-disease models

10 March 2021

12:00 p.m. ET

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Human genes associated with brain-related diseases are being discovered at an accelerating pace. A major challenge is to identify the mechanisms of action for these genes, and to determine potential therapeutic strategies. To elucidate these mechanisms in human cells, we established a CRISPR-based platform for genome-wide screens in human induced pluripotent stem cell (iPSC)-derived neurons, glia, and multi-lineage assembloids. Complex libraries of single-guide RNAs (sgRNAs) enable us to conduct genome-wide or focused loss-of-function (CRISPR interference, or CRISPRi) and gain-of-function screens (CRISPR activation, or CRISPRa). Such screens can reveal molecular players for phenotypes based on survival, stress resistance, fluorescent phenotypes, high-content imaging, and single-cell RNA-Seq. To uncover disease mechanisms and therapeutic targets, we are conducting genetic modifier screens for disease-relevant cellular phenotypes in patient-derived neurons and glia with familial mutations and isogenic controls. CRISPRi/a can also be used to model and functionally evaluate disease-associated changes in gene expression, such as those caused by expression quantitative trait locis (eQTLs), haploinsufficiency, or disease states of brain cells.

In this webinar, viewers will:

  • Learn about CRISPRi and CRISPRa-based functional genomics in iPSC models of brain disease
  • Discover CRISPR-based screens with single-cell RNA sequencing readouts
  • Gain insight into identification of disease mechanisms and therapeutic targets for brain disease through CRISPR screens
  • Have the opportunity to ask questions during the live broadcast.

This webinar will last for approximately 60 minutes.

Speaker bios

Martin Kampmann, Ph.D.

University of California, San Francisco
San Francisco, CA

Dr. Kampmann is an associate professor at the University of California, San Francisco (UCSF) Institute for Neurodegenerative Diseases and the Department of Biochemistry and Biophysics, and an investigator at the Chan Zuckerberg Biohub. He received his B.A. in biochemistry from Cambridge University and his Ph.D. in biophysics/cell biology from Rockefeller University. The goal of his research is to elucidate cellular mechanisms of brain disease and to develop new therapeutic strategies. He codeveloped the CRISPR interference and activation (CRISPRi and CRISPRa) screening technologies, and his lab has pioneered CRISPR-based functional genomics in cell types derived from induced pluripotent stem cells (iPSCs). A major focus is the investigation of neurodegenerative diseases in human iPSC-derived neurons, astrocytes, and microglia as well as 3D assembloids/organoids. Dr. Kampmann was named an NIH Director’s New Innovator, an Allen Distinguished Investigator, and he received the CZI Ben Barres Early Career Acceleration Award.

Jackie Oberst, Ph.D.

Washington, DC

Dr. Oberst did her undergraduate training at the University of Maryland, College Park, and her Ph.D. in Tumor Biology at Georgetown University, Washington D.C. She combined her interests in science and writing by pursuing an M.A. in Journalism from the Philip Merrill College of Journalism at the University of Maryland, College Park. Dr. Oberst joined Science/AAAS in 2016 as the Assistant Editor for Custom Publishing. Before then she worked at Nature magazine, the Howard Hughes Medical Institute, The Endocrine Society, and the National Institutes of Mental Health.

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