SC11: Genome Editing with CRISPR: Toward Novel Research, Translational, and Clinical Applications

SUNDAY, MARCH 1 | 5:30 - 8:30 PM (DINNER PROVIDED)

ABOUT THIS COURSE:

This main objective of this intensive three-hour-long course that has been compiled for specialists interested in applying genome editing technologies for both basic and translational research is to review the highlights and state-of-art applications of CRISPR/Cas9 gene editing technology. The course will review the basic aspects and mechanisms of CRISPR/Cas9, key molecular features of this technology, strengths and shortcomings of CRISPR genome editing systems, as well as most critical challenges in designing CRISPR experiments. Additional topics will cover more advanced facets of CRISPR application toolkit such as engineering disease models for in vivo research supporting preclinical drug development and employing CRISPR technology for diagnostic and therapeutic purposes.

TOPICS TO BE COVERED:

• Introduction into Genome Editing Technologies: from ZNF to TALEN to CRISPR/Cas9 to Base Editor Nucleases
• Non-Cas9-Based Systems for DNA and RNA Editing
• Synopsis of Various Delivery Systems and Available Gene Transfer Reagents to Support CRISPR in vivo and in vitro Technology Applications
• CRISPR Technologies for Pharmacologic Target Identification and Drug Screening
• Rational Design and Screening of CRISPR Libraries
• Development of Disease Models with CRISPR Technology for Translational Experimentation and Preclinical Drug Development
• Overview of Recently Introduced Clinical and Diagnostic Applications for CRISPR/Cas9 Technology

COURSE AGENDA:

5:30 pm Introduction to Genome Editing Technologies

Serguei Kozlov, PhD, MBA, PMP, Principal Scientist/PM, Team Leader PTO, Center for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research (NCI)

The introduction into genome editing technologies will cover ZNF to TALEN, CRISPR/Cas9, and base editor nucleases. We will then move on to discuss non-Cas9-based systems for DNA and RNA editing, and then offer a synopsis of various delivery systems and available gene transfer reagents to support CRISPR in vivo and in vitro technology applications. We’ll wrap up the first section with discussing CRISPR technologies for pharmacologic target identification and drug screening.

6:15 Dinner Buffet

6:45 CRISPR Technology from Design to Diagnostics

Serguei Kozlov, PhD, MBA, PMP, Principal Scientist/PM, Team Leader PTO, Center for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research (NCI)

This module will cover rational design and screening of CRISPR libraries, the development of disease models with CRISPR technology for translational experimentation and preclinical drug development. We’ll also provide an overview of recently introduced clinical and diagnostic applications for CRISPR/Cas9 technology and wrap up with a final discussion on tips and tricks and a Q&A session.

8:30 Course Ends

INSTRUCTOR:

Serguei Kozlov, PhD, MBA, PMP, Principal Scientist/PM, Team Leader PTO, 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 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 60 scientific papers, book chapters and original works in peer-reviewed journals.

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