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Ono Pharma Foundation Symposium has ended
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Session II: Hosted by Dr. David Corey [clear filter]
Monday, July 1
 

1:15pm EDT

Expanding the Scope of Base Editing
Through a combination of protein engineering and protein evolution, we recently developed two classes of base editors (CBE and ABE) that together enable all four types of transition mutations (C to T, T to C, A to G, and G to A) to be efficiently and cleanly installed or corrected at target positions in genomic DNA without making double-stranded DNA breaks. The four transition mutations collectively account for most known human pathogenic point mutations. Recently we have expanded the scope of base editing by enhancing its efficiency, product purity, targeting scope, and DNA specificity. By optimizing base editor expression, we developed “max” versions of cytosine and adenine base editors with greatly increase editing efficiency in mammalian cells.  To improve the targeting scope of base editing, we used our phage-assisted continuous evolution (PACE) system to rapidly evolve Cas9 and base editor variants with broadened PAM compatibility, higher DNA specificity, and enhanced editing capabilities. Finally, we are developing new base editor systems that operate on cellular RNA.

Speakers
avatar for David R. Liu, PhD

David R. Liu, PhD

Richard Merkin Professor, Director of the Merkin Institute of Transformative Technologies in Healthcare, and Vice-Chair, Harvard University
David R. Liu is the Richard Merkin Professor, Director of the Merkin Institute of Transformative Technologies in Healthcare, and Vice-Chair of the Faculty at the Broad Institute of Harvard and MIT; Thomas Dudley Cabot Professor of the Natural Sciences and Professor of Chemistry and... Read More →


Monday July 1, 2019 1:15pm - 1:45pm EDT
Abigail Adams Salon A/B

1:45pm EDT

Chemical biology of anti-cancer innate immunity
Speakers
avatar for Lingyin Li

Lingyin Li

Assistant Professor of Biochemistry, Stanford University
Dr. Li is an assistant professor in the Biochemistry Department and ChEM-H Institute at Stanford since 2015. Her lab works on understanding biochemical mechanisms of innate immunity and harnessing it to treat cancer. She majored in chemistry at University of Science and Technology... Read More →


Monday July 1, 2019 1:45pm - 2:15pm EDT
Abigail Adams Salon A/B

2:15pm EDT

Selective Neuronal Delivery of ASOs Targeting APP
Speakers
avatar for William Mobley, PhD

William Mobley, PhD

Professor, University of California, San Diego
Dr. Mobley is Associate Dean of Neurosciences Initiatives and Distinguished Professor of Neurosciences at UC San Diego. He serves as Executive Director of UCSD's Down Syndrome Center for Research and Treatment, and holds the Florence Riford Chair of Alzheimer Disease Research. He... Read More →


Monday July 1, 2019 2:15pm - 2:30pm EDT
Abigail Adams Salon A/B

2:30pm EDT

How druggable is the transcriptome? Finding small molecule binding sites in RNA.
Despite our increasing appreciation of non-coding RNA function, RNA has been conspicuously underrepresented in small-molecule drug development efforts until recently. Now it is clear binding pockets on RNA can make good drug targets, yet the properties and principles of small molecule binding sites on RNA remain poorly defined. I will discuss the challenges of defining small-molecule binding sites across the transcriptome and new approaches to address these challenges.

Speakers
avatar for Matthew Simon, PhD

Matthew Simon, PhD

Associate Professor of Molecular Biophysics and Biochemistry, Yale University
Matt grew up in Ann Arbor, MI and received his Ph.D. in Chemistry from UC Berkeley. He commuted between Berkeley and UCSF, working with Kevan Shokat developing chemical methods to make synthetic chromatin substrates to study the biochemistry of epigenetics. He continued this work... Read More →


Monday July 1, 2019 2:30pm - 2:45pm EDT
Abigail Adams Salon A/B

2:45pm EDT

Healthy Brains and Mixed Backbones
The central nervous system (CNS) is a very promising tissue for the use of antisense oligonucleotides (ASOs), with one drug approved and six others in clinical trials. Nevertheless, we have found that the administration of ASOs to the CNS often causes acute toxicity.  In this talk, we use a novel assay to quantify ASO-induced acute toxicity, explore its mechanism, and identify both modification and formulation approaches that provide ASOs with improved therapeutic index in both small (mouse) and large (sheep) brains. 

Speakers
avatar for Jonathan Watts, PhD

Jonathan Watts, PhD

Associate Professor, RNA Therapeutics Institute at UMass Medical School
Nucleic acid chemistry and chemical biology; gene silencing, gene activation and genome editing


Monday July 1, 2019 2:45pm - 3:00pm EDT
Abigail Adams Salon A/B