Keystone Symposia

Fairmont Empress Victoria / Victoria Conference Centre Floorplan

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This meeting took place in 2019

Here are the related meetings in 2021:
Precision Engineering of the Genome, Epigenome and Transcriptome (EK24)
Plant Genome Engineering: From Lab to Field (EK25)

For a complete list of the meetings for the upcoming/current season, see our meeting list, or search for a meeting.

Genome Engineering: From Mechanisms to Therapies (B4)

Organizer(s) Andrew May, Rodolphe Barrangou and Knut Woltjen
February 19—23, 2019
Fairmont Empress Victoria / Victoria Conference Centre • Victoria, BC Canada
Discounted Abstract Deadline: Oct 23, 2018
Abstract Deadline: Nov 20, 2018
Scholarship Deadline: Oct 23, 2018
Discounted Registration Deadline: Dec 19, 2018

Sponsored by BlueRock Therapeutics, Editas Medicine, Thermo Fisher Scientific Inc. and Vertex Pharmaceuticals Incorporated

Summary of Meeting:
The use of programmable nucleases such as CRISPR-Cas systems, ZFNs and TALENs has revolutionized cell biology by providing the ability to manipulate specific genetic and epigenetic states within living cells. These systems have been broadly applied as tools in research settings and increasingly are being developed to create improved models of disease and engineer cells for therapeutic purposes. Together with other DNA modifying systems such as recombinases, integrases and transposases, it is now possible to introduce mutations that will model human disease, build complex synthetic signaling networks to perform regulated functions and design cells to target specific disease states. Improvements to the methods involved requires understanding enzyme structures and mechanisms and how they intersect with cellular DNA repair systems. The intersection of this basic science with engineering approaches and improved cellular models is revolutionizing our understanding and treatment of human disease. The goal of this Keystone Symposium is to bring together people developing and studying genome engineering tools with groups who are applying them to build new disease models, identify disease mechanisms and drug targets, and develop cell-based therapeutics and genetic medicines. In addition to covering engineering of human and animal cells, this meeting will also highlight the emerging field of genome engineering to identify new anti-microbial and anti-viral drugs and applications towards next generation antibiotics. Invited talks will explore a broad range of topics covering new technologies, fundamental basic research, through the development of screening approaches, stem cell-based models of disease and design and development of cellular therapeutics.

View Scholarships/Awards
No registration fees are used to fund entertainment or alcohol at this conference

Conference Program    Print  |   View meeting in 12 hr (am/pm) time

The meeting will begin on Tuesday, February 19 with registration from 16:00 to 20:00 and a welcome mixer from 18:00 to 20:00. Conference events conclude on Saturday, February 23 with a closing plenary session from 17:00 to 19:30, followed by a social hour and entertainment. We recommend return travel on Sunday, February 24 in order to fully experience the meeting.


Arrival and Registration

Palm Court
Welcome Mixer
No registration fees are used to fund alcohol served at this function.

Palm Court



Palm Court
Welcome and Keynote Address

Salon A
* Andrew May, Sana Biotechnology, Inc., USA

Philip D. Gregory, Bluebird Bio, USA
Toward Therapeutic Genome Editing: A Brief History (And Some Lessons Learned from Classical Gene Therapy)

Genome Engineering Tools and Technologies

Salon A
* Dana Carroll, University of Utah School of Medicine, USA

Prashant Mali, University of California, San Diego, USA
Therapeutic Strategies via Genome Engineering: New Approaches and New Challenges

Coffee Break

Charles Gersbach, Duke University, USA
Epigenome Editing for Gene Therapy, Cell Programming and Functional Epigenomics

Alexis C. Komor, University of California, San Diego, USA
Using Uracil as a Genome Editing Intermediate

Peter Cameron, Caribou Biosciences, Inc., USA
Short Talk: Harnessing Type I CRISPR–Cas Systems for Human Genome Engineering

On Own for Lunch

Poster Setup

Salon B/C
Poster Viewing

Salon B/C
Workshop 1: Tools and Technologies

* Joanne Kamens, Addgene, USA

Abigail R. Lambert, Fred Hutchinson Cancer Research Center, USA
Structural and Functional Properties of Wild-Type and Engineered Meganucleases and MegaTALs

Henriette O'Geen, University of California, Davis, USA
Engineering Epigenetic Memory Requires Co-Targeting of Histone Methylatransferases and DNA Methylatransferases

Alister Funnell, Altius Institute for Biomedical Sciences, USA
Rapid Single-Cell Quantification of On- and Off-Target Nuclease Activity

Janin Grajcarek, Kyoto University, Japan
Identification of Microhomology-Flanked Deletion Mutations Across the Human Genome Enables Efficient Creation of Isogenic Disease Models in hiPSCs by CRISPR/Cas9

Hien Bao Dieu Thai, Korea Institute of Science and Technology, South Korea
DNAzymes-Based Tetrahedral Nanostructure for Enhanced Intracellular Gene-Silencing Activity

Brock Roberts, Allen Institute for Cell Science, USA
Systematic Gene Tagging to Illuminate Stem Cell Organization

Coffee Available

Prefunction 2
Structure and Mechanism of Genome Editing Systems

Salon A
* Maria Jasin, Memorial Sloan Kettering Cancer Center, USA

Benjamin P. Kleinstiver, Massachusetts General Hospital, USA
Engineered CRISPR Nucleases to Enhance Genome Editing

Osamu Nureki, University of Tokyo, Japan
Molecular Mechanism of CRISPR and Structure-Based Development of Genome Editing Tool toward Medical Applications

Edward J. Rebar, Sangamo Therapeutics, Inc., USA
Short Talk: Optimizing Nuclease Specificity for Gene Editing via Tuning of Cleavage Kinetics Enables Complete Gene Modification with No Detectable Off-Targets

Amit Choudhary, Harvard Medical School, USA
Short Talk: Synthetic Activators, Inhibitors and Degraders of CRISPR-Associated Nucleases

Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Salon B/C
Poster Session 1

Salon B/C



Palm Court
Poster Setup

Salon B/C
Poster Viewing

Salon B/C
Engineered Models of Genetic Disease

Salon A
* Bruce R. Conklin, University of California, San Francisco, USA

Danwei Huangfu, Memorial Sloan Kettering Cancer Institute, USA
Human Pluripotent Stem Cells as a Genetic Model for Human Development and Disease

Knut Woltjen, CiRA, Kyoto University, Japan
Precise Human Disease Allele Creation and Correction through Microhomology-Mediated End Joining

Coffee Break

Amy J. Wagers, Harvard University, USA
Gene Editing in Stem Cells

Erika Sasaki, Central Institute for Experimental Animals, Japan
Development of Genetically Modified Non-Human Primate Disease Models

Diogo Mosqueira, University of Nottingham, UK
Short Talk: CRISPR/Cas9 Genome Editing in Human Pluripotent Stem Cell-Cardiomyocytes to Model and Treat Hypertrophic Cardiomyopathy


Salon B/C
Poster Session 2

Salon B/C
Coffee Available

Prefunction 2
Design and Engineering of Cellular Devices

Salon A
* Alexis C. Komor, University of California, San Diego, USA

Wilson Wong, Boston University, USA
Mammalian Cell Design Using Synthetic Biology

Timothy K. Lu, Massachusetts Institute of Technology, USA
Synthetic Gene Circuits for Next-Generation Therapeutics

Zoltan Ivics, Paul Ehrlich Institute, Germany
Transposons: Molecular Parasites Tamed for Advanced Genome Engineering

Yale S. Michaels, University of Oxford, UK
Short Talk: A Generalizable Method for Precisely Tuning Gene Expression Levels in Mammalian Cells with Engineered MicroRNA Target Sites

On Own for Dinner



Palm Court
Genome Editing Screens for Function and Disease Mechanisms

Salon A
* Danwei Huangfu, Memorial Sloan Kettering Cancer Institute, USA

Fyodor D. Urnov, University of California, Berkeley, USA
Editing Human Genome Control Circuits to Reveal Disease Mechanisms and Targets for Intervention in the Clinic

Jan E. Carette, Stanford University, USA
CRISPR-Cas Screens for Studying Virus-Host Interactions

Coffee Break

Nozomu Yachie, University of Tokyo, Japan
Tracing Dynamics of Cells and Molecules using DNA Barcodes and Genome Editing

Britt S. Adamson, Princeton University, USA
Mapping the Processes of Genome Editing with High-Resolution Functional Genomics

Take the Bull by the Horns: Steps to a Fulfilling Career in Science

* Joanne Kamens, Addgene, USA

On Own for Lunch

Coffee Available

Prefunction 2
Harnessing DNA Repair Mechanisms for Genome Engineering

Salon A
* Britt S. Adamson, Princeton University, USA

Maria Jasin, Memorial Sloan Kettering Cancer Center, USA
Homologous Recombination and End-Joining Mechanisms in Genome Editing

Beeke Wienert, University of California, San Francisco, USA
Unbiased Detection of CRISPR Off-Targets in vivo using DISCOVER-Seq

Nancy Maizels, University of Washington School of Medicine, USA
Gene Correction at Targeted DNA Breaks

Andrew May, Sana Biotechnology, Inc., USA
DNA Repair Outcomes Provide Insight into Genome Editing Mechanisms in Primary Cell Systems

Tetsushi Sakuma, Hiroshima University, Japan
Short Talk: Concurrent MMEJ-Assisted Fusional Knock-In of Long Gene Cassette in Human Cells

Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Salon C



Palm Court
Genome Engineering of Bacteria for Therapeutic and Diagnostic Applications

Salon A
* Andrew May, Sana Biotechnology, Inc., USA

Rodolphe Barrangou, North Carolina State University, USA
Engineering Lactobacilli for Human Health Applications

Jason M. Peters, University of Wisconsin-Madison, USA
Bacterial CRISPRi Screens to Identify the Mode of Action of Novel Antibiotics

Coffee Break

Richard P. Novick, New York University, USA
Conversion of Staphylococcal Pathogenicity Islands to CRISPR-Cas9-Based Antibacterial Drones

Joel Berry, Caribou Biosciences, USA
Short Talk: Utilizing CRISPR-Based Genome Editing for Microbiome Engineering

Akos Nyerges, Hungarian Academy of Sciences, Hungary
Short Talk: Predicting Antibiotic Resistance by Targeted Mutagenesis and Directed Evolution in Pathogenic Bacteria

David R. Edgell, University of Western Ontario, Canada
Short Talk: High Efficiency Inter-Species Conjugative Transfer of a CRISPR Nuclease for Targeted Bacterial Elimination

On Own for Lunch

Workshop 2: Delivery Methods
A collection of short talks chosen from the abstracts describing methods and approaches for in vitro and in vivo delivery of genome engineering technologies. The workshop would cover topics including devices for electroporation, mechanical introduction of molecules into cells, cell-specific targeting, viruses and viral-like particles, nanoparticles to address the key need to develop delivery vehicles for effective use of genome engineering approaches in therapeutics and research models.

Salon A
* Shondra M. Pruett-Miller, St. Jude Children’s Research Hospital, USA

Dana V. Foss, University of California, Berkeley, USA
Engineering Cas9 for T-Cell Specific Uptake and Therapeutic Genome Editing

Jacquelyn L S Hanson, SQZ Biotechnologies, USA
Microfluidic Delivery of Bioactive Molecules via SQZ Platform Enables Efficient T Cell Genome Engineering with Preserved Functionality

Lauren Elizabeth Woodard, Vanderbilt University, USA
Less Is More: How Less Recombinase Expression Produces More Genome-Modified Cells

Taisuke Kato, Niigata University, Japan
Gene Therapy for DRPLA Model Mice by AAV-Delivered CRISPR / Cas9

Eric Aird, University of Minnesota, USA
Enhancing HDR Efficiency by Tethering DNA to Cas9 via a Fused HUH Endonuclease

Erin Morgan, University of California, Santa Barbara, USA
Controlling the Genome: Light-Activated Delivery of Gene Editing Proteins and siRNA Allows for Up and Down Regulation of the Genome using Hollow Gold Nanoparticles

Masato Ohtsuka, Tokai University, Japan
i-GONAD: A Method for Generation of Genome-Edited Rodents without ex vivo Handling of Embryos

Anthony L. Forget, Intellia Therapeutics, USA
Supra-Therapeutic Levels of Transgene Expression Achieved in vivo by CRISPR/Cas9 Mediated Targeted Gene Insertion

Coffee Available

Prefunction 2
Genome Editing for Treating Human Disease

Salon A
* Fyodor D. Urnov, University of California, Berkeley, USA

Bruce R. Conklin, University of California, San Francisco, USA
Using Patient-Derived iPSC Tissues to Model Precise Genome Surgery

Leonela Amoasii, Exonics Therapeutics, USA
Gene Editing Restores Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy

Lukas Jeker, University of Basel, Switzerland
Short Talk: Repairing Foxp3 Mutations in T Cells Restores Regulatory T Cell Function

Jorge Mansilla-Soto, Memorial Sloan Kettering Cancer Center, USA
Advancing CAR-T Therapy with Precise Genome Engineering

Vic E. Myer, Atlas Ventures, USA
Controlling Rearrangement Frequencies in the Context of Multigene Genome Editing

Meeting Wrap-Up: Outcomes and Future Directions (Organizers)

Salon A
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.

Palm Court/Crystal Ballroom
Entertainment is not subsidized by conference registration fees nor any U.S. federal government grants. Funding for this expense is provided by other revenue sources.

Palm Court/Crystal Ballroom



*Session Chair †Invited, not yet responded.

We gratefully acknowledge support for this conference from:

Directors' Fund

These generous unrestricted gifts allow our Directors to schedule meetings in a wide variety of important areas, many of which are in the early stages of research.

Click here to view all of the donors who support the Directors' Fund.

Keystone Symposia thanks our Sponsors(s) for generously supporting this meeting:

BlueRock Therapeutics Editas Medicine
Thermo Fisher Scientific Inc. Vertex Pharmaceuticals Incorporated

We gratefully acknowledge additional support from these exhibitors at this conference:

Agilent Technologies, Inc. BEX CO., LTD.
Horizon Discovery MilliporeSigma
Please stop by to meet these exhibitors during the conference.

We gratefully acknowledge additional support for this conference from:

American Society of Gene & Cell Therapy (ASGCT) Integrated DNA Technologies

We gratefully acknowledge additional in-kind support for this conference from those foregoing speaker expense reimbursements:

bluebird bio

We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:

CRISPR Journal, published by Mary Ann Liebert, Inc. publishers

Special thanks to the following for their support of Keystone Symposia initiatives to increase participation at this meeting by scientists from underrepresented backgrounds:

Click here to view more of these organizations

If your organization is interested in joining these entities in support of Keystone Symposia, please contact: Sarah Lavicka, Director of Corporate Relations, Email:,
Phone:+1 970-262-2690

Click here for more information on Industry Support and Recognition Opportunities.

If you are interested in becoming an advertising/marketing in-kind partner, please contact:
Nick Dua, Senior Director, Communications, Email:,
Phone:+1 970-262-1179