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|Systematic Systematic endogenous gene tagging with CRISPR/Cas9 in human iPSCs; lessons learned|
|Tanya Grancharova, Amanda Haupt, and Brock Roberts, The Allen Institute|
|A workflow and media system optimization for successful editing in stem cells|
|Rhonda Newman, Thermo Fisher Scientific|
|A pharmaceutical industry perspective on using CRISPR gene editing and iPSCs|
|Stuart Chambers, Amgen|
|Genome editing and neural differentiation on human iPS cells derived from patients with Parkinson’s Disease|
|Xin Jiang, Yumanity|
|Panel Discussion / Round Table|
|Moderators: Brock Roberts, The Allen Institute|
Ru Gunawardane, Director, Stem Cells and Gene Editing, Allen Institute for Cell Science
Rhonda Newman, Senior Staff Scientist, Thermo Fisher Scientific
• Hope, Ambiguity, & the Imminence of “The Miracle Factor”
• The Need For Curated Information In The Public And Academic Communities
a) The Diagnosis Dilemma (Phenotype) and public / patient inferences
b) Redefinition Of “The Miracle Factor”
• Stem Cells 2.0—How CRISPR / Stem Cell Treatments Can Help Tame “The Wild West”
Doug Oliver, Founder-Chair, Regenerative Medicine
Doug is a writer, editorial contributor, and patient advocate from Nashville, Tennessee. He holds a Master’s Degree in Social Work from the University of New England and has specialized in work on health care policy, compliance, and clinical case advocacy.
He is also a stem cell patient. At age 32, he was diagnosed with macular degeneration, a disease that attacks the retina, causing blindness of central vision. Doug became legally blind at age 45. In August 2015, he underwent autologous bone-marrow stem cell therapy to both eyes, and regained much of his lost vision. He received his driver’s license 5 months later. Doug’s story has reached millions through international broadcasts, interviews with the national press, and countless affiliate television and news media outlets.
The advent of CRISPR-Cas gene editing allows for generation of knockout mutations in disease relevant genes in mammalian cells with relative ease. Organoids are complex, multicellular structures that recapitulate key aspects of an organ, such as cellular organization and function. CRISPR gene editing has been applied to organoid systems to generate cellular disease models, including modeling colorectal cancer in intestinal organoids and diseases of nephron segments in kidney organoids.
Genome engineered organoids have myriad applications for biomedical research and show great promise for future translational applications, including regenerative therapies in disease. The general workflow for editing stem cells with CRISPR will be discussed, with a focus on generating knockouts of disease relevant genes and its applications for disease modeling in human organoids.
The workshop will cover the following topics:
• The general workflow for editing stem cells with CRISPR, with a focus on generating knockout mutations in disease relevant genes.
• Disease modeling in stem cell-derived organoids and its applications for biomedical research.
• Discussion of technical considerations and potential translational applications of genome engineered human organoids
Nelly Cruz, Research Scientist, University of Washington