Delivery: A Major Challenge for Gene Editing and Gene Therapy
By Robert Waymouth (PhD ‘87)
Price increases to $22 because of Baylands. Price includes lunch, tax and gratuity
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With the advancement of new gene editing strategies (CRISPR/Cas9, TALEN), new tools and therapies are emerging, but the delivery of genes to the appropriate cells and tissues in-vivo remains a formidable challenge. Just like packaging, delivery logistics is extremely important.
In this talk, I will describe our recent scientific breakthroughs to precisely deliver genes into living animals. Functional delivery of messenger RNA (mRNA) in-vivo is key to implementing potentially transformative strategies for vaccination, protein replacement therapy and genome editing, as mRNA carries the instructions for cells to generate their own therapeutics.
We have developed an expedient synthesis of a new, tunable and effective class of synthetic biodegradable materials for mRNA delivery: charge-altering releasable transporters (CARTs).
CART delivery has been used to protect mice from cancer, to cure established tumors in mice and to effect gene editing by both knock in and knock out of protein function. This technology is effective against a variety of cell types and can be targeted to various organs.
CARTs are structurally unique and operate through an unprecedented mechanism, serving initially as oligo(α-amino ester) cations that complex, protect and deliver mRNA, and then change physical properties through a degradative, charge-neutralizing intramolecular rearrangement, leading to intracellular release of functional mRNA and highly efficient protein expression. CART delivery works for siRNA, mRNA, pDNA, LNA, and coformulations involving CRISPR/Cas 9.
About Our Speaker
Robert is the Robert Eckles Swain Professor of Chemistry at Stanford University. He received B.S. in Mathematics and B.A. in Chemistry from Washington and Lee University and his Ph.D. in Chemistry at the Caltech in 1987 with Professor R.H. Grubbs. He was a postdoctoral fellow with the late Professor Piero Pino at the ETH in Zurich in 1987 and joined the faculty at Stanford as an Assistant Professor in 1988. He received the Alan T. Waterman Award from the NSF in 1996, the Cooperative Research Award in Polymer Science in 2009, and EPA's Presidential Green Chemistry Challenge Award in 2012 with Dr. James Hedrick.
He has won several university teaching awards, including the Walter J. Gores Award, the Phi Beta Kappa Teaching Award, and is currently a Bass Fellow in Undergraduate Education.
His research interests are at the interface of Inorganic, Organic and Polymer Chemistry, in particular the development of new concepts in catalysis for the selective synthesis of both macromolecules and fine chemicals. Particular areas of interest include catalytic polymerization reactions, selective oxidation catalysis, the development of organocatalytic polymerization strategies, and the design of functional macromolecules for applications in biology and medicine.