June 12-14, 2018
Seattle, WA

Register by THIS Friday May 18 to save up to $700

Day One
Wednesday, June 13th 2018

Day Two
Thursday, June 14th 2018

08.30
Morning Coffee & Refreshments

09.00
Chair’s Opening Remarks

A Continued Discussion of Using CRISPR for Regulating the Genome

09.10
Enhancer Switching Regulates Sox2 During the Transition From Embryonic to Neural Stem Cells

Synopsis

• CRISPR-mediated enhancer deletion shows the enhancers required to regulate Sox2 in embryonic and neural stem cells are different
• Chromatin architecture is altered co-incident with the switch in enhancer usage
• Transcription factors bind intergenic regions in ES cells that are poised to regulate Sox2 in neural stem cells

09.40
Elucidating Disease Mechanisms & Therapeutic Targets in Human IPSC Models by CRISPR-Based Functional Genomics

Synopsis

• We have established genetic screens based on CRISPR, CRISPR interference (CRISPRi) and CRISPR activation (CRISPRa) in human iPSC-derived cell types
• Genetic modifier screens in patient-derived iPSCs and isogenic CRISPR-corrected lines can reveal mechanisms of disease-associated genes and therapeutic strategies
• We have established cellular phenotypes relevant for neurodegenerative diseases in human iPSC-derived neurons, astrocytes and microglia and conducted first genetic modifier screens in a pooled format
• We are also establishing a platform for CRISPR-based arrayed high-content screens in iPSC-derived cells to investigate complex and non-cell autonomous phenotypes

10.10
Combining Patient-Derived IPSCs & CRISPR Technology in Disease Modeling & Drug Discovery for Rare Genetic Disorders

Synopsis

• “Cluster-and-conquer” approach for >80 rare human diseases linked to the intermediate filament gene family
• CRISPR/Cas9 gene editing of Alexander Disease and Giant Axonal Neuropathy patient iPSCs to obtain isogenic control cells
• 2D and 3D differentiation platforms using disease mutant and isogenic control iPSCs to generate astrocytes and neurons
• Validation of disease targets and testing of small molecule compounds in iPSCsderived astrocytes

10.40
Morning Refreshments

11.10
A Scaled Framework for CRISPR Editing of Human Pluripotent Stem Cells to Study Psychiatric Disease

Synopsis

• We report a standardized CRISPR-Cas9 approach with robust benchmarking at each step to edit a large set of psychiatric disease-implicated genes in hPSCs
• Transcriptional state and nucleosome positioning around targeted loci are not correlated with CRISPR editing efficiency
• Editing frequencies vary between different hPSC lines and correlate with genomic stability
• Precision editing with small and large ssDNAs provides an efficient platform for future editing frameworks

11.40
Synthetic sgRNA Enables Highly Efficient & Consistent CRISPR Editing of Cells for Automation, Cell Engineering & Therapeutic Applications

Synopsis

• Achieving consistent and high editing efficiencies with CRISPR is critical for automation, cell engineering and therapeutic applications with primary cells, and remains a significant challenge
• Through a collaborative effort, we demonstrate that use of synthetic sgRNA for CRISPR yields improved and consistent editing efficiencies that are required for such applications

11.55
Gene Repair in Human Hematopoietic Stem Cells

  • Jenny Shin Jacob Corn’s Lab Postdoctoral Researcher, Jacob Corn’s Lab Innovative Genomics Institute UC Berkeley

Synopsis

• Mechanisms of HDR in human cells
• Challenges of editing in hematopoietic stem cells
• Progress towards translation of gene editing therapies for sickle cell disease

12.25
Lunch & Networking

13.25
Using CRISPR to Illuminate Organs & Organoids

Synopsis

– Kidney organoids derived from Allen Institute iPS cell lines reveal organelle dynamics at the tissue scale

– CRISPR can be used to knock out whole organelles in human tissues

– Using CRISPR in embryos allows the growth of entire organs from a different species in mice

13.55
Human IPSC-Based Disease Modeling Reveals a Retrotransposon With Repeat Expansion as the Causal Mechanism of X-Linked Dystonia Parkinsonism

Synopsis

• Human iPSC-based XDP disease modeling reveals causative mutation of XDP
• An XDP-specific SVA insertion induces intron retention and down-regulation of TAF1
• CRISPR/Cas9 excision of SVA rescues aberrant splicing and cTAF1 expression in XDP
• Expression profiling implicates neurodevelopment and dystonia pathways in XDP iPSCderived neural cells

14.25
CRISPRing iPSCs to Efficiently Model Genetic Diseases

Synopsis

• Generating iPSC from patients with genetic disorders
• Creating isogenic iPSCs for disease modeling with the CRISPR/Cas9 system
• Applying the CRISPR/Cas9 system to Cerebral Cavernous Malformation (CCM) patientspecific iPSCs to generate knockout models
• Developing 3D culture platforms of iPSCs-derived endothelial cells for transcriptome and proteomic studies

14.55
Afternoon Refreshments

15.25
Round table sessions

Synopsis

16.25
Chair’s Closing Remarks

16.30
Close of CRISPR Stem Cell Congress 2018