CRISPR/Cas9 cell therapeutics – The next generation of cures

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Hosted by GEN, Produced with Support from Lonza

The discovery of CRIPSR/Cas9 as simple and effective tool for targeted modification of cells has opened up new possibilities for gene or cell therapies. We will discuss strategies for using CRIPSR/Cas9 for curing genetic diseases, and the impact on next generation disease model systems and drug screening.
 

Topics discussed: 

• Introduction to CRISPR/Cas9
• Application examples with focus on use for sickle cell disease
• Opportunities and challenges for the use of CRISPR/Cas9 in gene/cell therapeutic applications
• Use of CRISPR for next generation disease model systems
• Non-viral transfer of CRISPR/Cas9 cargos and up-scaling of the transfection process

Speakers:

• Matt Porteus, MD, PhD, Associate Professor of Pediatrics, Stanford University
• Greg Alberts, PhD, Global Subject Matter Expert Transfection and Genome Editing, Lonza Bioscience Solutions

CRISPR transfection: 

• Efficient genome editing using Lonza's Nucleofector™ Technology

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