Cambridge Healthtech 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.

TS3A: Genome Editing with Target Nucleases: Theory and Practice of CRISPR/Cas9 Technology Applications from Basic Research to Precision Therapies

The Westin Copley Place | Boston, MA
Tuesday, June 19 - Wednesday, June 20
Day 1: 9:00 am – 5:00 pm | Day 2: 9:00 am – 12:00 pm

Great Republic

Serguei Kozlov, PhD, MBA, PMP, Principal Scientist/PM, Team Leader PTO, Center for Advanced Preclinical Research, Frederick National Laboratory for Cancer Research (NCI)
Danilo Maddalo, PhD, Lab Head, ONC Pharmacology, Novartis Institutes for BioMedical Research, Novartis Pharma AG

This rigorous day and a half program is ideal for discovery and translational scientists who are looking for a balanced knowledge of gene editing applications in drug discovery and development. Taught by experts in the CRISPR/Cas9 field, this program was designed as a succinct introduction into the technology “nuts and bolts”. Beginning from describing the basic aspects, key molecular features, strengths and shortcomings of CRISPR/Cas9 technology, the workshop instructors will advance towards sharing in-depth knowledge touching upon all facets of state-of-art genome editing applications, e.g. constructing of experimental cell culture based systems, engineering disease in vivo models supporting preclinical drug development workflows, rational design and functional screening of sgRNA libraries, and many others. Instructors will furthermore strive to achieve a balance between presenting theory information and conducting hands-on exercises in exploring available digital frameworks for designing and resourcing CRISPR/Cas9 studies, as well as troubleshooting complex experimental scenarios and conducting Q&A sessions.

Topics to be Covered in the Seminar:

  • Introduction into Genome Editing Technologies: from ZNF to TALEN to CRISPR/Cas9
    • Biology of dsDNA Break Repair and Its Implications for CRISPR Experimental Designs
    • Immunogenicity of CRISPR Reagents: Experimental Considerations
    • nCas9 and dCas9 Isoforms and Their Applications (CRISPRa, CRISPRi, precision epigenetic, chromosome localization, single base editor nucleases, etc.)
  • Non-Cas9 Based Systems for DNA and RNA Editing
  • CRISPR/Cas9 Bioinformatics and Experimental Design Resources
  • Overview of CRISPR Delivery Systems and Reagent Collections Available to Support CRISPR Technology Applications
  • CRISPR Technologies in Target Identification and Drug Screening
  • Functional Screening of CRISPR Libraries
  • Development of Preclinical Models with CRISPR Technology
  • Emerging Clinical Applications of CRISPR/Cas9 Technology
  • Final Discussion/Workshop Adjourn

Instructor Biographies:

Serguei KozlovSerguei 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 of generating diverse preclinical murine models (including genetically engineered mouse (GEM) models, GEM-derived syngeneic allograft (GDA) models, patient-derived (PDX) and conventional cell lines-based xenograft 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 GEM vs. GDA vs. PDX mouse models. Oversee from day one launch and operations of the NCI 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.

Danilo MaddaloDanilo Maddalo, PhD, Lab Head, ONC Pharmacology, Novartis Institutes for BioMedical Research, Novartis Pharma AG
Danilo Maddalo obtained his PhD in biochemistry and molecular biology from the University of Karlsruhe (Germany). He thereafter joined Memorial Sloan Kettering Cancer Center (New York, USA) as a research fellow where he generated a CRISPR-induced mouse model of oncogenic chromosomal rearrangements. He finally moved to Novartis (Basel, Switzerland) where he currently leads a laboratory focusing on pharmacology and generation of preclinical models.

Training Seminar Information

Each CHI 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 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 engage in track hopping, as to not disturb the hands-on style instruction being offered to the other participants.


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