The Do's and Do Not's of CRISPR-Cas9-based Gene Targeting in Human Blood Stem and Effector Cells

Sponsored By Synthego

Originally aired September 27th, 2018

The CRISPR-Cas9 system enables targeted manipulations of the genome and has revolutionized biomedical research. We have learned in recent years that gene disruption and gene insertion are possible with high frequencies even in challenging cell types. Using Cas9 protein in combination with chemically-modified sgRNAs has enabled the efficient generation of genetic knockout and knock-ins in therapeutically relevant human cells like hematopoietic stem cells, primary T cells, and others. Matthew Porteus’ Laboratory at Stanford University; the Porteus Lab focuses on developing new genome editing tools for therapeutic applications. In this webinar, two members of the Porteus lab share insights about development of these tools and ongoing pre-clinical work on treatment of hematologic diseases like sickle cell anemia and immunodeficiencies. They also discuss how they are exploring the beneficial attributes of the editing system to engineer cells of the immune system for immunotherapy to develop next-generation cell therapies.

A live Q&A session will follow the presentations, offering you a chance to pose questions to our expert panelists.
Daniel Dever, PhD
Research Instructor
Stanford University
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Volker Wiebking, MD
Postdoctoral Research Fellow, Stem Cell Transplantation
Stanford Medicine
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Moderator: Kevin Davies, PhD
Executive Editor
The CRISPR Journal

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