Day One
Monday 13th November, 2017

Day Two
Tuesday 14th November, 2017

Registration & Coffee

Chair’s Opening Remarks

Applications of Precision Gene Editing Technology in Biomedical Research

Cellular Indel Profiles & Dynamics Induced by Different CRISPR/Cas9 Delivery Formats: Applying Ex Vivo Methodologies In Vivo


  • Plasmid-, piggyback-, lenti- and RNP -Cas9 and gRNA delivery methodologies
  • Cas9 indel formation detection methodologies: Pro’s and con’s of the available methods
  • Indel formation dynamics and profiles induced by various CRISPR/Cas9 delivery methods

Detection of Genome Editing Outcomes by Droplet Digital™ PCR

  • Yuichiro Miyaoka Project Leader, Regenerative Medicine Project, Tokyo Metropolitan Institute of Medical Science


  • Development and optimisation of genome editing methods for a sensitive, rapidreadout tool for edit validation and off-target detection
  • Droplet Digital PCR (ddPCR) enables sensitive (<0.1%), precise absolute quantification of NHEJ and HDR alleles in a rapid, high-throughput format

Learning From CRISPR/Cas9: Advancing Bioinformatics for Precision Gene Editing

  • David Parry-Smith Group Leader, Stem Cell Informatics , Wellcome Trust Sanger Institute


  • An informatics perspective on genome editing with CRISPR/Cas9
  • Improving on-target and off-target computational analysis for designing genome editing experiments
  • Examples of feedback from high-throughput experiments to guide learning from data and development of novel algorithms

Speed Networking & Morning Refreshments

Precise Genome Editing of Hematopoietic Stem Cells

  • Rasmus Bak Assistant Professor, Group Leader, Aarhus University

Hit Identification & Confirmation Strategies in Arrayed CRISPR-Cas9 Screening


  • Execution of a synthetic crRNA library screen using high content imaging identify potential hits important in the cell cycle
  • Statistical examination of six phenotypic parameters reveal robust, functional knockout for multiple reagents per gene
  • Follow-up experiments including confirmation of phenotype in an independent experiment, expression analysis, and editing efficiency will be discussed
  • Gene annotation and crRNA target position analysis give novel insights into system biology
  • Confirmation of hits with orthogonal reagents (siRNA) supports high-confidence hits
  • Strategies for further validation and hit stratification will be discussed

Custom CRISPR Screens Targeting Subsets of Genes

Networking Lunch

Development of Advanced Disease Models Through Precision Gene Editing

CRISPR/Cas9 Genome Editing in Mice

  • Lin Wu Director, Genome Modification Facility , Harvard University


  • Based on analysis of the current data, the efficiency for gene KO through NHEJ has been significantly higher (~50%) than gene KI through HDR or HR (~15%)
  • Reagents of different forms of Cas9 and gRNA used for gene editing
  • Targeted gene disruption and deletion to generate gene KO mice
  • Site-specific transgene integration to generate gene KI mice

Modeling of Neurodegenerative Diseases in a Dish with the Help of CRISPR-Cas9

  • Kristine Freude Associate Professor, Group of Stem Cells & Embryology, Department , of Veterinary & Animal Sciences, University of Copenhagen


  • Challenges and possibilities for CRISPR-Cas9 gene editing in cellular models of neurodegeneration
  • Gene specific hurdles for CRISP-Cas9 gene editing
  • Example of a disease model for frontotemporal dementia linked to chromosome 3

Employing the CRISPR/Cas9 System in Pre-Clinical Testing of T cell Receptor-Based Bispecific Drug Candidates

  • Dan Blat Senior Scientist- Manager , Immunocore


  • Overview of the ImmTAC platform: TCR-based bispecific biologics for cancer immunotherapy
  • The unique requisites when deleting/editing HLA presented peptides in cell lines
  • Our current yet ever changing CRISPR workflow
  • Discussion of some of the technical details and observations we have made throughout our CRISPR work

Afternoon Refreshments & Poster Session

CRISPR Roundtables – Advancing CRISPR Technology to the Next Level


  1. Harnessing CRISPR to understand disease mechanisms in in vivo models
    Pedro Costa, Sir Henry Wellcome Postdoctoral Fellow, King’s College London
  2. Modulating epigenetic factors using CRISPR gene editing
  3. Harnessing CRISPRi or CRISPRA for interference or augmentation of gene expression as a drug discovery tool
  4. Developing human iPS cells for therapeutic development of monoclonal antibodies

Close of Day 1