The ARVO/Alcon Keynote Series is supported by the ARVO Foundation through a contribution from Alcon Laboratories.
Monday, April 30
5:30pm - 6:45pm
Jennifer Doudna, PhD - CRISPR-Cas Gene Editing: Biology, Technology and Ethics
HHMI Investigator, Li Ka Shing Chancellor's Chair in Biomedical and Health Sciences
Professor, Departments of Molecular & Cell Biology and Chemistry, UC Berkeley
Executive Director, Innovative Genomics Institute
As an internationally renowned professor of Chemistry and Molecular and Cell Biology at U.C. Berkeley, Doudna and her colleagues rocked the research world in 2012 by describing a simple way of editing the DNA of any organism using an RNA-guided protein found in bacteria. This technology, called CRISPR-Cas9, has opened the floodgates of possibility for human and non-human applications of gene editing, including assisting researchers in the fight against HIV, sickle cell disease and muscular dystrophy. Doudna is an Investigator with the Howard Hughes Medical Institute and a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors and the American Academy of Arts and Sciences. She is also a Foreign Member of the Royal Society, and has received many other honors including the Breakthrough Prize in Life Sciences, the Heineken Prize, the BBVA Foundation Frontiers of Knowledge Award and the Japan Prize. She is the co-author with Sam Sternberg of “A Crack in Creation,” a personal account of her research and the societal and ethical implications of gene editing.
Gene editing with CRISPR technology is transforming biology. Understanding the underlying chemical mechanisms of RNA-guided DNA and RNA cleavage provides a foundation for both conceptual advances and technology development. Doudna will discuss how bacterial CRISPR adaptive immune systems inspire creation of powerful genome engineering tools, enabling advances in both fundamental biology and applications in medicine. She will also discuss the ethical challenges of some of these applications.
Get a glimpse of Doudna: Watch her Ted Talk: How CRISPR lets us edit our DNA.
Thursday, May 3
2:15 - 3:45pm
Shoukhrat Mitalipov, PhD - Principles of gene repair in human embryos
Director, Center for Embryonic Cell and Gene Therapy of Oregon Health & Science University (OHSU)
Professor, Division of Reproductive Developmental Sciences at Oregon National Primate Research Center
Shoukhrat Mitalipov is director of the Center for Embryonic Cell and Gene Therapy of Oregon Health & Science University (OHSU). He is also a professor in the Division of Reproductive & Developmental Sciences at Oregon National Primate Research Center, OHSU. Mitalipov earned his PhD degree in Developmental & Stem Cell Biology from Research Center for Medical Genetics, in Moscow, Russia. He came to Utah State University in 1995 to conduct his postdoctoral research in stem cell and developmental biology and moved to OHSU in 1998. Mitalipov’s research interest is to understand the mechanisms of cytoplasmic control of nuclear genome identity and reprogramming of somatic cells to the totipotent and pluripotent states. Another objective is to develop novel germline gene therapy approaches for the treatment of inherited human diseases. Mitalipov is known for his leading discoveries in producing human patient-matched embryonic stem cells using somatic cell nuclear transfer. His team has also pioneered a gene therapy approach that prevents transmission of genetic defects in mitochondrial genes from mothers to their children.
In vitro fertilization (IVF) represents a successful cell therapy approach for treatment of infertility. However, IVF is increasingly utilized for preimplantation genetic diagnosis (PGD) followed by embryo selection to prevent the transmission of heritable human diseases. Our Center is actively investigating novel germ line gene therapy approaches that would allow to repair gene defects in mutant gametes or early preimplantation embryos. We are focused on answering important safety and efficacy questions regarding techniques that could one day be useful in preventing thousands of inherited genetic disorders that affect millions of people worldwide. The focus of this lecture is to inform on applications of gene editing and gene replacement strategies in preclinical and clinical studies demonstrating feasibility, efficacy and long-term safety of germ line gene therapy.
Get a glimpse of Mitalipov: Watch as he discussses his research.