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 SHORT COURSE 4: GENOME EDITING USING CRISPR

Sunday, January 18 | 5:00-8:00 pm  

Mammalian cells are the workhorses for biopharmaceutical production. Thus, genome engineering/editing of these hosts to improve product quality and yields are of great interest. CRISPR, the newest gene editing tool, is gaining popularity among protein engineers and cell line developers. This course provides an introduction to CRISPR technology and insights on implementation for your protein expression and production pipeline.

Detailed Agenda 

5:00 pm Welcome and Introductions

5:10 Co-Presentations by Instructors Helene Faustrup Kildegaard, Norman Garceau and Deepak Reyon

5:55 Dinner Break

6:15 Co-Presentations by Instructors (cont’d)

7:45 Interactive Q&A with Instructors and Participants

8:00 Close of Short Course

 

Presentations:

Genome Editing in CHO Cells Using CRISPR Cas9 

Helene Faustrup Kildegaard, Ph.D., Co-Principal Investigator, Novo Nordisk Foundation Center for Biosustainability, Technical University of Denmark 

Chinese hamster ovary (CHO) cells are widely used for the production of therapeutic proteins. Thus genome engineering of CHO cells for improved product quality and yield is of great interest. At this course, our hands-on experience with the CRISPR Cas9 technology for generating gene disruptions and targeted gene insertions in CHO cells will be presented together with the bioinformatics tool “CRISPy” for rapid sgRNA target selection.

Introduction to CRISPR-Cas9 Engineered Nucleases for Genome Editing

Norman Garceau, Ph.D., CSO, Blue Sky Biotech, Inc.

The CRISPR-Cas9 engineered nuclease is an enabling technology that is revolutionizing genetics and has the potential transform medicine. A historical overview of CRISPR-Cas9 and its applications will be presented.

Generation of Custom Cell Lines Using CRISPR RNA-Guided Cas9 Nuclease

Deepak Reyon, Ph.D., Scientist I, Genetically Engineered Model Center, Biogen Idec

Custom cell lines are a vital workhorse both for basic research and drug development. The advent of the easily customizable and highly efficient CRISPR-Cas9 system has greatly reduced the time and effort required to generate such cell lines. Although certain constraints remain, they can be overcome to a large extent using different strategies that will be addressed in this talk.

 

Speaker Biographies: 

Helene Faustrup KildegaardHelene Faustrup Kildegaard, Ph.D., Co-Principal Investigator, Novo Nordisk Foundation Center for Biosustainability, Technical University of Denmark 

Helene Faustrup Kildegaard is Co-Principal Investigator at the Novo Nordisk Foundation Center for Biosustainability at Technical University of Denmark. She received her Ph.D. degree in cellular and molecular biology from the Danish Cancer Society/Technical University of Denmark. She was then awarded a postdoc grant on therapeutic protein production in CHO from the Lundbeck Foundation and the Technical University of Denmark. Her current research focuses on applying  -omics data and developing efficient genome editing tools to engineer CHO cell factories for increased production and improved quality of therapeutic proteins.

Norman GarceauNorman Garceau, Ph.D., CSO, Blue Sky Biotech, Inc.

Dr. Norman Garceau is the Chief Scientific Officer at Blue Sky BioServices and is responsible for overseeing all science-related activities and for ensuring that scientific excellence is at the forefront of the company. Prior to joining Blue Sky, Dr. Garceau was employed at Pfizer Global Research and Development where he served in a variety of roles in spanning molecular and cellular biology, protein production and small molecule discovery. Dr. Garceau earned a Ph.D. in Biochemistry from Dartmouth Medical School where he studied the molecular basis of circadian rhythmicity in Neurospora crassa. Subsequently, he pursued postdoctoral research at Pfizer and Dartmouth Medical School, studying the molecular basis of signal transduction for members of the TNF receptor family.

Deepak ReyonDeepak Reyon, Ph.D., Scientist I, Genetically Engineered Model Center, Biogen Idec

My research interests focus on exploring and implementing different strategies, using precise targetable nucleases, for high-throughput genome engineering in various model organisms and cell lines. Over the years I have been involved in developing methods for high-throughput assembly and validation of: zinc fingers nucleases (ZFNs), known as context-dependent assembly - CoDA (Sander, et al, Nature Methods 2011); transcription activator-like effector nucleases (TALENs), known as fast ligation-based automatable solid-phase high-throughput (FLASH) assembly (Reyon, et al, Nature Biotechnology 2012); clustered regularly interspaced palindromic repeats (CRISPR) RNA-guided CRISPR-associated (Cas9) proteins (Hwang, et al, Nature Biotechnology 2013).


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