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This meeting took place in 2019
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Genome Engineering: From Mechanisms to Therapies (B4)
Organizer(s) Andrew May, Rodolphe Barrangou and Knut Woltjen
February 19—23, 2019
Fairmont Empress Victoria • Victoria, British Columbia 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
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
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.
TUESDAY, FEBRUARY 19
WEDNESDAY, FEBRUARY 20
THURSDAY, FEBRUARY 21
FRIDAY, FEBRUARY 22
SATURDAY, FEBRUARY 23
SUNDAY, FEBRUARY 24
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.
TUESDAY, FEBRUARY 19
18:00—20:00
Welcome Mixer
No registration fees are used to fund alcohol served at this function.
08:30—09:30
Welcome and Keynote Address
Meeting has ended...abstracts no longer viewable online.
*
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)
Toward Therapeutic Genome Editing: A Brief History (And Some Lessons Learned from Classical Gene Therapy)
09:30—11:45
Genome Engineering Tools and Technologies
Meeting has ended...abstracts no longer viewable online.
*
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
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
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
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
Short Talk: Harnessing Type I CRISPR–Cas Systems for Human Genome Engineering
14:30—16:30
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
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
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
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
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
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
Systematic Gene Tagging to Illuminate Stem Cell Organization
17:00—19:00
Structure and Mechanism of Genome Editing Systems
Meeting has ended...abstracts no longer viewable online.
*
Maria Jasin,
Memorial Sloan Kettering Cancer Center, USA
Benjamin P. Kleinstiver,
Massachusetts General Hospital, USA
Engineered CRISPR Nucleases to Enhance Genome Editing
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
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
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
Short Talk: Synthetic Activators, Inhibitors and Degraders of CRISPR-Associated Nucleases
19:00—20:00
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.
08:30—11:30
Engineered Models of Genetic Disease
Meeting has ended...abstracts no longer viewable online.
*
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
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
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
Gene Editing in Stem Cells
Erika Sasaki,
Central Institute for Experimental Animals, Japan
Development of Genetically Modified Non-Human Primate Disease Models
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
Short Talk: CRISPR/Cas9 Genome Editing in Human Pluripotent Stem Cell-Cardiomyocytes to Model and Treat Hypertrophic Cardiomyopathy
15:00—17:00
Design and Engineering of Cellular Devices
Meeting has ended...abstracts no longer viewable online.
*
Alexis C. Komor,
University of California, San Diego, USA
Timothy K. Lu,
Massachusetts Institute of Technology, USA
Synthetic Gene Circuits for Next-Generation Therapeutics
Synthetic Gene Circuits for Next-Generation Therapeutics
Zoltan Ivics,
Paul Ehrlich Institute, Germany
Transposons: Molecular Parasites Tamed for Advanced Genome Engineering
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
Short Talk: A Generalizable Method for Precisely Tuning Gene Expression Levels in Mammalian Cells with Engineered MicroRNA Target Sites
08:30—11:30
Genome Editing Screens for Function and Disease Mechanisms
Meeting has ended...abstracts no longer viewable online.
*
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
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
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
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
Mapping the Processes of Genome Editing with High-Resolution Functional Genomics
11:30—12:30
Take the Bull by the Horns: Steps to a Fulfilling Career in Science
Meeting has ended...abstracts no longer viewable online.
*
Joanne Kamens,
Addgene, USA
17:00—19:15
Harnessing DNA Repair Mechanisms for Genome Engineering
Meeting has ended...abstracts no longer viewable online.
*
Britt S. Adamson,
Princeton University, USA
Maria Jasin,
Memorial Sloan Kettering Cancer Center, USA
Homologous Recombination and End-Joining Mechanisms in Genome Editing
Homologous Recombination and End-Joining Mechanisms in Genome Editing
Beeke Wienert,
University of California, Berkeley, USA
Unbiased Detection of CRISPR Off-Targets in vivo using DISCOVER-Seq
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
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
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
Short Talk: Concurrent MMEJ-Assisted Fusional Knock-In of Long Gene Cassette in Human Cells
19:15—20:15
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.
08:30—11:30
Genome Engineering of Bacteria for Therapeutic and Diagnostic Applications
Meeting has ended...abstracts no longer viewable online.
*
Andrew May,
Sana Biotechnology, Inc., USA
Rodolphe Barrangou,
North Carolina State University, USA
Engineering Lactobacilli for Human Health Applications
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
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
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
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
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
Short Talk: High Efficiency Inter-Species Conjugative Transfer of a CRISPR Nuclease for Targeted Bacterial Elimination
14:30—16:30
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.
*
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
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
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
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
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
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
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
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
Supra-Therapeutic Levels of Transgene Expression Achieved in vivo by CRISPR/Cas9 Mediated Targeted Gene Insertion
17:00—19:15
Genome Editing for Treating Human Disease
Meeting has ended...abstracts no longer viewable online.
*
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
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
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
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
Advancing CAR-T Therapy with Precise Genome Engineering
Vic E. Myer,
Editas Medicine, USA
Controlling Rearrangement Frequencies in the Context of Multigene Genome Editing
Controlling Rearrangement Frequencies in the Context of Multigene Genome Editing
19:15—19:30
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
Meeting has ended...abstracts no longer viewable online.
19:30—20:30
Social Hour with Lite Bites
No registration fees are used to fund alcohol served at this function.
20:00—23:00
Entertainment
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.
*Session Chair †Invited, not yet responded.
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