Cambridge Healthtech Institute Training Seminars offer real-life case studies, problems encountered and solutions applied, and extensive coverage of the basic science underlying each topic. Experienced Training Seminar instructors offer a mix of formal lectures, interactive discussions and activities to help attendees maximize their learning experiences. These immersive trainings will be of value to scientists from industry and academic research groups who are entering new fields – and to those working in supporting roles that will benefit from an in-depth briefing on a specific aspect of the industry.
TS7C: Genome Editing with CRISPR: Towards Novel Research, Translational and Clinical Applications
thursday, September 3 - friday, September 4
DAY 1: THURSDAY
1:40 - 5:20 pm Seminar Sessions
DAY 2: FRIDAY
8:30 am - 3:40 pm Seminar Sessions
12:30 - 1:30 Lunch Provided
Exhibit Hall Refreshment Breaks also provided.
Instructor:
Serguei Kozlov, PhD, MBA, PMP, Principal Scientist/PM, Team Leader Preclinical Technology and Optimization Team, Center for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research (NCI)
Introduction:
This rigorous day and a half instruction seminar is compiled for specialists interested in in-depth learning and subsequent employing of various genome editing technologies for both basic and translational research. The presented material will comprehensively
cover the state-of-art information on gene editing strategies and applications in various areas, such as in vitro and in vivo disease modelling, functional genomic analyses, drug discovery and development, diagnostic and therapeutic areas. Beginning
from discussion of introductory level basic technology aspects, key molecular features, capabilities and pitfalls, strengths and shortcomings of currently available CRISPR/Cas9 systems, the instructor will advance towards sharing more specific information
related to virtually all facets of present-day genome editing applications, such as constructing of cell culture based experimental systems, engineering precision targeting disease models for in vivo research, applying CRISPR methodologies to support
preclinical drug development workflows, rational design and functional screening of sgRNA libraries, application of CRISPR/Cas9 technology for clinical purposes and many others. Instructors will strive to achieve a balance between presenting theory
information and conducting some practical tasks, e.g. in exploring available digital resources for designing and enabling CRISPR/Cas9 studies, as well as assist in troubleshooting specific complex experimental scenarios and conduct extensive expertise
exchange and Q&A sessions.
Key Topics to be Covered During the Seminar:
- Introduction into Genome Editing Technologies: from ZNF’s and TALEN’s to CRISPR/Cas9, non-Cas9 based CRISPR systems and Base Editor Nucleases
- Specific Mechanistic Aspects of CRISPR Technology, Including:
- Processes of dsDNA Break Repair and Their Implications for CRISPR Experimental Designs
- Immunogenicity of CRISPR Reagents: Experimental Considerations
- nCas9 and dCas9-“nuclease-null” Isoforms of Editing Nucleases and Their Applications (CRISPRa, CRISPRi, precision epigenetic, chromosome localization, single base editor nucleases, etc.)
- Non-S.pyogenes Cas9 Based Systems for DNA and RNA Editing
- CRISPR/Cas9 Bioinformatics and Experimental Design Resources
- Synopsis of Various Delivery Systems and Available Reagent Collections to Support CRISPR In Vivo and In Vitro Technology Applications
- CRISPR Technologies in Target Identification and Drug Screening
- Rational Design and Screening of CRISPR Libraries
- Development of Disease Models with CRISPR Technology for Translational Experimentation or Preclinical Drug Development
- Overview of Emerging Clinical and Diagnostic Applications for CRISPR/Cas9 Technology
- Final Discussion/’Tips and Tricks’ Session
- Troubleshooting Specific Cases and Workshop Adjourn
Agenda:
Day One, Thursday May 7
- Introduction
- Basic Principles of Genome Editing
- CRISPR Technology Special Topics 1
- CRISPR Technology Special Topics II
- Applications of CRISPR Technology in Preclinical Disease Modeling
- Methodologies Involving Nuclease-Null (dCas9)
Fusion Proteins
Day Two, Friday May 8
- Therapeutic and Diagnostic Applications of CRISPR
- Designing and Screening sgRNA Libraries
- Immunogenicity and Delivery of CRISPR Reagents in vivo
About the Instructor:
Serguei Kozlov, PhD, MBA, PMP, Principal Scientist/PM, Team Leader Preclinical Technology and Optimization Team, Center for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research (NCI)
Internationally recognized expert in advanced methodologies for developing genetically and biologically engineered preclinical murine disease models for studies of the molecular mechanisms of carcinogenesis, tumor immune response, immune surveillance,
and inflammation, mechanisms of drug resistance, implication of cancer stem cells, etc.; and to explore such models for preclinical drug development, disease systems analyses and biomarker discovery. Initiated, supervised, and successfully conducted
multiple research projects and independent large-scale research and development programs aimed at investigation of molecular mechanisms governing carcinogenesis, biomarker discovery and preclinical drug evaluation in various mouse models. Oversees
the preclinical technology development operations at the Frederick National Laboratory Center for Advanced Preclinical Research – an innovative preclinical cancer modeling and drug development initiative based on genetically engineered murine
models – with senior management responsibilities. Co-authored over 70 scientific papers, book chapters and original works in peer-reviewed journals.
What is a Training Seminar?
Each Training Seminar offers 1.5 Days of instruction with start and stop times for each day shown above and on the Event-at-a-Glance published in the onsite Program & Event Guide. Training Seminars will include morning and afternoon refreshment breaks,
as applicable, and lunch will be provided to all registered attendees on the full day of the class.
Each person registered specifically for the training seminar will be provided with a hard copy handbook for the seminar in which they are registered. A limited number of additional handbooks will be available for other delegates who wish to attend the
seminar, but after these have been distributed no additional books will be available.
Though CHI encourages track and symposia hopping between conference programs, we ask that Training Seminars not be disturbed once they have begun. In the interest of maintaining the highest quality learning environment for Training Seminar attendees,
and because Seminars are conducted differently than conference programming, we ask that attendees commit to attending the entire program, and NOT engaging in track hopping, as to not disturb the hands-on style instruction being offered to the other
participants.
