WEBINAR: Pioneering the Possible


Richard A Morgan, Ph.D.
Editas Medicine, USA | Senior Vice President, Immunogenetics


Emerging gene-editing technologies are nearing a revolutionary phase in genetic medicine by precisely modifying or repairing causal genetic defects. Genome editing using RNA-guided nuclease technology, such as the CRISPR/Cas system, has gained widespread attention for its potential to improve current cell and gene therapies. This may include any number of DNA sequence manipulations, such as knocking out a deleterious gene, introducing a particular mutation, or directly repairing a defective sequence by site-specific recombination. This lecture will present key aspects of the CRISPR/Cas system describing how these technologies truly unlock the full potential of genome editing and how a rigorous approach to specificity is key to clinical applications. I will describe our ex vivo applications in sickle cell disease as well as our program on in vivo gene editing for Leber Congenital Amaurosis Type 10 (CLA10), an early-onset retinal degeneration disease caused by mutations in the CEP290 gene. The LCA10 program will be the first in human direct in vivo application of CRISPR/Cas technology.

Learn more about Sartorius solutions for CAR-T Discovery & Development at www.sartorius.com/car-t-research.

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