Web Desc
Genome Engineering: From Mechanisms to Therapies
Organizer(s): Andrew May, Rodolphe Barrangou and Knut Woltjen
Date: February 19 - 23, 2019
Location: Fairmont Empress Victoria / Victoria Conference Centre, Victoria, BC, Canada
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.
Scholarship Deadline: October 23 2018
Discounted Abstract Deadline: October 23 2018
Abstract Deadline: November 20 2018
Discounted Registration Deadline: December 19 2018
Keystone Symposia thanks our Sponsor(s) for generously supporting this meeting:
BlueRock TherapeuticsEditas MedicineThermo Fisher Scientific Inc.Vertex Pharmaceuticals Incorporated
We gratefully acknowledge additional support from these exhibitors at this conference:
Agilent Technologies, Inc.BEX CO., LTD.Horizon DiscoveryMilliporeSigmaSynthego
We gratefully acknowledge additional support for this conference from:
AddgeneAmerican 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
$10,000 - $24,999
Mary Ann Liebert, Inc. Publishers
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
We appreciate the organizations that provide Keystone Symposia with additional support, such as marketing and advertising:

Click here to view more of these organizations
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

Program

Tuesday, February 19 | 4:00PM - 8:00PM
Arrival and Registration
Room: Palm Court


Tuesday, February 19 | 6:00PM - 8:00PM
Welcome Mixer
Room: Palm Court
No registration fees are used to fund alcohol served at this function.

Wednesday, February 20 | 7:30AM - 8:30AM
Breakfast
Room: Palm Court


Wednesday, February 20 | 8:30AM - 9:30AM
Welcome and Keynote Address
Room: Salon A

Speaker 1 of 2
* Andrew May, Sana Biotechnology, Inc., USA

Wednesday, February 20 | 8:30AM - 9:30AM
Welcome and Keynote Address
Room: Salon A

Speaker 2 of 2
Philip D. Gregory, Bluebird Bio, USA
Toward Therapeutic Genome Editing: A Brief History (And Some Lessons Learned from Classical Gene Therapy)

Wednesday, February 20 | 9:30AM - 11:45AM
Genome Engineering Tools and Technologies
Room: Salon A

Speaker 1 of 6
* Dana Carroll, University of Utah School of Medicine, USA

Wednesday, February 20 | 9:30AM - 11:45AM
Genome Engineering Tools and Technologies
Room: Salon A

Speaker 2 of 6
Prashant Mali, University of California, San Diego, USA
Therapeutic Strategies via Genome Engineering: New Approaches and New Challenges

Wednesday, February 20 | 9:30AM - 11:45AM
Genome Engineering Tools and Technologies
Room: Salon A


Coffee Break

Wednesday, February 20 | 9:30AM - 11:45AM
Genome Engineering Tools and Technologies
Room: Salon A

Speaker 4 of 6
Charles Gersbach, Duke University, USA
Epigenome Editing for Gene Therapy, Cell Programming and Functional Epigenomics

Wednesday, February 20 | 9:30AM - 11:45AM
Genome Engineering Tools and Technologies
Room: Salon A

Speaker 5 of 6
Alexis C. Komor, University of California, San Diego, USA
Using Uracil as a Genome Editing Intermediate

Wednesday, February 20 | 9:30AM - 11:45AM
Genome Engineering Tools and Technologies
Room: Salon A

Speaker 6 of 6
Peter Cameron, Caribou Biosciences, Inc., USA
Short Talk: Harnessing Type I CRISPR–Cas Systems for Human Genome Engineering

Wednesday, February 20 | 11:45AM - 5:00PM
On Own for Lunch


Wednesday, February 20 | 11:45AM - 1:00PM
Poster Setup
Room: Salon B/C


Wednesday, February 20 | 1:00PM - 10:00PM
Poster Viewing
Room: Salon B/C


Wednesday, February 20 | 2:30PM - 4:30PM
Workshop 1: Tools and Technologies

Speaker 1 of 7
* Joanne Kamens, Addgene, USA

Wednesday, February 20 | 2:30PM - 4:30PM
Workshop 1: Tools and Technologies

Speaker 2 of 7
Abigail R. Lambert, Fred Hutchinson Cancer Research Center, USA
Structural and Functional Properties of Wild-Type and Engineered Meganucleases and MegaTALs

Wednesday, February 20 | 2:30PM - 4:30PM
Workshop 1: Tools and Technologies

Speaker 3 of 7
Henriette O'Geen, University of California, Davis, USA
Engineering Epigenetic Memory Requires Co-Targeting of Histone Methylatransferases and DNA Methylatransferases

Wednesday, February 20 | 2:30PM - 4:30PM
Workshop 1: Tools and Technologies

Speaker 4 of 7
Alister Funnell, Altius Institute for Biomedical Sciences, USA
Rapid Single-Cell Quantification of On- and Off-Target Nuclease Activity

Wednesday, February 20 | 2:30PM - 4:30PM
Workshop 1: Tools and Technologies

Speaker 5 of 7
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

Wednesday, February 20 | 2:30PM - 4:30PM
Workshop 1: Tools and Technologies

Speaker 6 of 7
Hien Bao Dieu Thai, Korea Institute of Science and Technology, South Korea
DNAzymes-Based Tetrahedral Nanostructure for Enhanced Intracellular Gene-Silencing Activity

Wednesday, February 20 | 2:30PM - 4:30PM
Workshop 1: Tools and Technologies

Speaker 7 of 7
Brock Roberts, Allen Institute for Cell Science, USA
Systematic Gene Tagging to Illuminate Stem Cell Organization

Wednesday, February 20 | 4:30PM - 5:00PM
Coffee Available
Room: Prefunction 2


Wednesday, February 20 | 5:00PM - 7:00PM
Structure and Mechanism of Genome Editing Systems
Room: Salon A

Speaker 1 of 5
* Maria Jasin, Memorial Sloan Kettering Cancer Center, USA

Wednesday, February 20 | 5:00PM - 7:00PM
Structure and Mechanism of Genome Editing Systems
Room: Salon A

Speaker 2 of 5
Benjamin P. Kleinstiver, Massachusetts General Hospital, USA
Engineered CRISPR Nucleases to Enhance Genome Editing

Wednesday, February 20 | 5:00PM - 7:00PM
Structure and Mechanism of Genome Editing Systems
Room: Salon A

Speaker 3 of 5
Osamu Nureki, University of Tokyo, Japan
Molecular Mechanism of CRISPR and Structure-Based Development of Genome Editing Tool toward Medical Applications

Wednesday, February 20 | 5:00PM - 7:00PM
Structure and Mechanism of Genome Editing Systems
Room: Salon A

Speaker 4 of 5
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

Wednesday, February 20 | 5:00PM - 7:00PM
Structure and Mechanism of Genome Editing Systems
Room: Salon A

Speaker 5 of 5
Amit Choudhary, Harvard Medical School, USA
Short Talk: Synthetic Activators, Inhibitors and Degraders of CRISPR-Associated Nucleases

Wednesday, February 20 | 7:00PM - 8:00PM
Social Hour with Lite Bites
Room: Salon B/C
No registration fees are used to fund alcohol served at this function.

Wednesday, February 20 | 7:30PM - 10:00PM
Poster Session 1
Room: Salon B/C


Thursday, February 21 | 7:30AM - 8:30AM
Breakfast
Room: Palm Court


Thursday, February 21 | 8:00AM - 8:30AM
Poster Setup
Room: Salon B/C


Thursday, February 21 | 8:30AM - 5:00PM
Poster Viewing
Room: Salon B/C


Thursday, February 21 | 8:30AM - 11:30AM
Engineered Models of Genetic Disease
Room: Salon A

Speaker 1 of 7
* Bruce R. Conklin, University of California, San Francisco, USA

Thursday, February 21 | 8:30AM - 11:30AM
Engineered Models of Genetic Disease
Room: Salon A

Speaker 2 of 7
Danwei Huangfu, Memorial Sloan Kettering Cancer Institute, USA
Human Pluripotent Stem Cells as a Genetic Model for Human Development and Disease

Thursday, February 21 | 8:30AM - 11:30AM
Engineered Models of Genetic Disease
Room: Salon A

Speaker 3 of 7
Knut Woltjen, CiRA, Kyoto University, Japan
Precise Human Disease Allele Creation and Correction through Microhomology-Mediated End Joining

Thursday, February 21 | 8:30AM - 11:30AM
Engineered Models of Genetic Disease
Room: Salon A


Coffee Break

Thursday, February 21 | 8:30AM - 11:30AM
Engineered Models of Genetic Disease
Room: Salon A

Speaker 5 of 7
Amy J. Wagers, Harvard University, USA
Gene Editing in Stem Cells

Thursday, February 21 | 8:30AM - 11:30AM
Engineered Models of Genetic Disease
Room: Salon A

Speaker 6 of 7
Erika Sasaki, Central Institute for Experimental Animals, Japan
Development of Genetically Modified Non-Human Primate Disease Models

Thursday, February 21 | 8:30AM - 11:30AM
Engineered Models of Genetic Disease
Room: Salon A

Speaker 7 of 7
Diogo Mosqueira, University of Nottingham, UK
Short Talk: CRISPR/Cas9 Genome Editing in Human Pluripotent Stem Cell-Cardiomyocytes to Model and Treat Hypertrophic Cardiomyopathy

Thursday, February 21 | 11:30AM - 12:30PM
Lunch
Room: Salon B/C


Thursday, February 21 | 12:00PM - 2:30PM
Poster Session 2
Room: Salon B/C


Thursday, February 21 | 2:30PM - 3:00PM
Coffee Available
Room: Prefunction 2


Thursday, February 21 | 3:00PM - 5:00PM
Design and Engineering of Cellular Devices
Room: Salon A

Speaker 1 of 5
* Alexis C. Komor, University of California, San Diego, USA

Thursday, February 21 | 3:00PM - 5:00PM
Design and Engineering of Cellular Devices
Room: Salon A

Speaker 2 of 5
Wilson Wong, Boston University, USA
Mammalian Cell Design Using Synthetic Biology

Thursday, February 21 | 3:00PM - 5:00PM
Design and Engineering of Cellular Devices
Room: Salon A

Speaker 3 of 5
Timothy K. Lu, Massachusetts Institute of Technology, USA
Synthetic Gene Circuits for Next-Generation Therapeutics

Thursday, February 21 | 3:00PM - 5:00PM
Design and Engineering of Cellular Devices
Room: Salon A

Speaker 4 of 5
Zoltan Ivics, Paul Ehrlich Institute, Germany
Transposons: Molecular Parasites Tamed for Advanced Genome Engineering

Thursday, February 21 | 3:00PM - 5:00PM
Design and Engineering of Cellular Devices
Room: Salon A

Speaker 5 of 5
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

Thursday, February 21 | 5:00PM - 5:00PM
On Own for Dinner


Friday, February 22 | 7:30AM - 8:30AM
Breakfast
Room: Palm Court


Friday, February 22 | 8:30AM - 11:30AM
Genome Editing Screens for Function and Disease Mechanisms
Room: Salon A

Speaker 1 of 6
* Danwei Huangfu, Memorial Sloan Kettering Cancer Institute, USA

Friday, February 22 | 8:30AM - 11:30AM
Genome Editing Screens for Function and Disease Mechanisms
Room: Salon A

Speaker 2 of 6
Fyodor D. Urnov, University of California, Berkeley, USA
Editing Human Genome Control Circuits to Reveal Disease Mechanisms and Targets for Intervention in the Clinic

Friday, February 22 | 8:30AM - 11:30AM
Genome Editing Screens for Function and Disease Mechanisms
Room: Salon A

Speaker 3 of 6
Jan E. Carette, Stanford University, USA
CRISPR-Cas Screens for Studying Virus-Host Interactions

Friday, February 22 | 8:30AM - 11:30AM
Genome Editing Screens for Function and Disease Mechanisms
Room: Salon A


Coffee Break

Friday, February 22 | 8:30AM - 11:30AM
Genome Editing Screens for Function and Disease Mechanisms
Room: Salon A

Speaker 5 of 6
Nozomu Yachie, University of Tokyo, Japan
Tracing Dynamics of Cells and Molecules using DNA Barcodes and Genome Editing

Friday, February 22 | 8:30AM - 11:30AM
Genome Editing Screens for Function and Disease Mechanisms
Room: Salon A

Speaker 6 of 6
Britt S. Adamson, Princeton University, USA
Mapping the Processes of Genome Editing with High-Resolution Functional Genomics

Friday, February 22 | 11:30AM - 12:30PM
Take the Bull by the Horns: Steps to a Fulfilling Career in
Science


Speaker 1 of 1
* Joanne Kamens, Addgene, USA

Friday, February 22 | 11:30AM - 5:00PM
On Own for Lunch


Friday, February 22 | 4:30PM - 5:00PM
Coffee Available
Room: Prefunction 2


Friday, February 22 | 5:00PM - 7:15PM
Harnessing DNA Repair Mechanisms for Genome Engineering
Room: Salon A

Speaker 1 of 6
* Britt S. Adamson, Princeton University, USA

Friday, February 22 | 5:00PM - 7:15PM
Harnessing DNA Repair Mechanisms for Genome Engineering
Room: Salon A

Speaker 2 of 6
Maria Jasin, Memorial Sloan Kettering Cancer Center, USA
Homologous Recombination and End-Joining Mechanisms in Genome Editing

Friday, February 22 | 5:00PM - 7:15PM
Harnessing DNA Repair Mechanisms for Genome Engineering
Room: Salon A

Speaker 3 of 6
Beeke Wienert, University of California, San Francisco, USA
Unbiased Detection of CRISPR Off-Targets in vivo using DISCOVER-Seq

Friday, February 22 | 5:00PM - 7:15PM
Harnessing DNA Repair Mechanisms for Genome Engineering
Room: Salon A

Speaker 4 of 6
Nancy Maizels, University of Washington School of Medicine, USA
Gene Correction at Targeted DNA Breaks

Friday, February 22 | 5:00PM - 7:15PM
Harnessing DNA Repair Mechanisms for Genome Engineering
Room: Salon A

Speaker 5 of 6
Andrew May, Sana Biotechnology, Inc., USA
DNA Repair Outcomes Provide Insight into Genome Editing Mechanisms in Primary Cell Systems

Friday, February 22 | 5:00PM - 7:15PM
Harnessing DNA Repair Mechanisms for Genome Engineering
Room: Salon A

Speaker 6 of 6
Tetsushi Sakuma, Hiroshima University, Japan
Short Talk: Concurrent MMEJ-Assisted Fusional Knock-In of Long Gene Cassette in Human Cells

Friday, February 22 | 7:15PM - 8:15PM
Social Hour with Lite Bites
Room: Salon C
No registration fees are used to fund alcohol served at this function.

Saturday, February 23 | 7:30AM - 8:30AM
Breakfast
Room: Palm Court


Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A

Speaker 1 of 8
* Andrew May, Sana Biotechnology, Inc., USA

Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A

Speaker 2 of 8
Rodolphe Barrangou, North Carolina State University, USA
Engineering Lactobacilli for Human Health Applications

Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A

Speaker 3 of 8
Jason M. Peters, University of Wisconsin-Madison, USA
Bacterial CRISPRi Screens to Identify the Mode of Action of Novel Antibiotics

Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A


Coffee Break

Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A

Speaker 5 of 8
Richard P. Novick, New York University, USA
Conversion of Staphylococcal Pathogenicity Islands to CRISPR-Cas9-Based Antibacterial Drones

Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A

Speaker 6 of 8
Joel Berry, Caribou Biosciences, USA
Short Talk: Utilizing CRISPR-Based Genome Editing for Microbiome Engineering

Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A

Speaker 7 of 8
Akos Nyerges, Hungarian Academy of Sciences, Hungary
Short Talk: Predicting Antibiotic Resistance by Targeted Mutagenesis and Directed Evolution in Pathogenic Bacteria

Saturday, February 23 | 8:30AM - 11:30AM
Genome Engineering of Bacteria for Therapeutic and Diagnosti
c Applications

Room: Salon A

Speaker 8 of 8
David R. Edgell, University of Western Ontario, Canada
Short Talk: High Efficiency Inter-Species Conjugative Transfer of a CRISPR Nuclease for Targeted Bacterial Elimination

Saturday, February 23 | 11:30AM - 5:00PM
On Own for Lunch


Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 1 of 9
* Shondra M. Pruett-Miller, St. Jude Children’s Research Hospital, USA

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 2 of 9
Dana V. Foss, University of California, Berkeley, USA
Engineering Cas9 for T-Cell Specific Uptake and Therapeutic Genome Editing

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 3 of 9
Jacquelyn L S Hanson, SQZ Biotechnologies, USA
Microfluidic Delivery of Bioactive Molecules via SQZ Platform Enables Efficient T Cell Genome Engineering with Preserved Functionality

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 4 of 9
Lauren Elizabeth Woodard, Vanderbilt University, USA
Less Is More: How Less Recombinase Expression Produces More Genome-Modified Cells

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 5 of 9
Taisuke Kato, Niigata University, Japan
Gene Therapy for DRPLA Model Mice by AAV-Delivered CRISPR / Cas9

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 6 of 9
Eric Aird, University of Minnesota, USA
Enhancing HDR Efficiency by Tethering DNA to Cas9 via a Fused HUH Endonuclease

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 7 of 9
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

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 8 of 9
Masato Ohtsuka, Tokai University, Japan
i-GONAD: A Method for Generation of Genome-Edited Rodents without ex vivo Handling of Embryos

Saturday, February 23 | 2:30PM - 4:30PM
Workshop 2: Delivery Methods
Room: Salon A
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.
Speaker 9 of 9
Anthony L. Forget, Intellia Therapeutics, USA
Supra-Therapeutic Levels of Transgene Expression Achieved in vivo by CRISPR/Cas9 Mediated Targeted Gene Insertion

Saturday, February 23 | 4:30PM - 5:00PM
Coffee Available
Room: Prefunction 2


Saturday, February 23 | 5:00PM - 7:15PM
Genome Editing for Treating Human Disease
Room: Salon A

Speaker 1 of 6
* Fyodor D. Urnov, University of California, Berkeley, USA

Saturday, February 23 | 5:00PM - 7:15PM
Genome Editing for Treating Human Disease
Room: Salon A

Speaker 2 of 6
Bruce R. Conklin, University of California, San Francisco, USA
Using Patient-Derived iPSC Tissues to Model Precise Genome Surgery

Saturday, February 23 | 5:00PM - 7:15PM
Genome Editing for Treating Human Disease
Room: Salon A

Speaker 3 of 6
Leonela Amoasii, Exonics Therapeutics, USA
Gene Editing Restores Dystrophin Expression in a Canine Model of Duchenne Muscular Dystrophy

Saturday, February 23 | 5:00PM - 7:15PM
Genome Editing for Treating Human Disease
Room: Salon A

Speaker 4 of 6
Lukas Jeker, University of Basel, Switzerland
Short Talk: Repairing Foxp3 Mutations in T Cells Restores Regulatory T Cell Function

Saturday, February 23 | 5:00PM - 7:15PM
Genome Editing for Treating Human Disease
Room: Salon A

Speaker 5 of 6
Jorge Mansilla-Soto, Memorial Sloan Kettering Cancer Center, USA
Advancing CAR-T Therapy with Precise Genome Engineering

Saturday, February 23 | 5:00PM - 7:15PM
Genome Editing for Treating Human Disease
Room: Salon A

Speaker 6 of 6
Vic E. Myer, Atlas Ventures, USA
Controlling Rearrangement Frequencies in the Context of Multigene Genome Editing

Saturday, February 23 | 7:15PM - 7:30PM
Meeting Wrap-Up: Outcomes and Future Directions (Organizers)
Room: Salon A


Saturday, February 23 | 7:30PM - 8:30PM
Social Hour with Lite Bites
Room: Palm Court/Crystal Ballroom
No registration fees are used to fund alcohol served at this function.

Saturday, February 23 | 8:00PM - 11:00PM
Entertainment
Room: 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.

Saturday, February 23 | 8:00PM - 11:00PM
Cash Bar
Room: Palm Court/Crystal Ballroom


Sunday, February 24 | 10:24AM - 10:24AM
Departure


*Session Chair.