Kavli Research & Enterprise Discussions (K.R.E.D)
Our Kavli Research and Enterprise Discussion (K.R.E.D) will be given by Krzysztof Palczewski.
Krzysztof Palczewski is a Member of the National Academy of Sciences and the National Academy of Medicine.
In 2012 he was awarded Prize of the Foundation for Polish Science, the most prestigious scientific award for Polish scientists, for characterizing crystal structures of native and activated G protein-coupled receptor, rhodopsin, involved in eyesight.
His MS and PhD, are from the University of Wroclaw and Technical University of Wroclaw, respectively (Poland). His early posts were at the University of Florida and the Oregon Health Sciences University. Dr. Palczewski completed much of his pivotal research at the University of Washington. In 2005 he moved to become the Chair and John H. Hord professor of Pharmacology at Case Western Reserve University. In 2018 he joined University of California, Irvine, where he is a Distinguished Professor and is leading the Center for Translational Vision Research.
Krzysztof's talk is entitled "Precise Genome Editing in the Eye"
The abstract is below.
Gene augmentation and CRISPR/Cas9 genome editing techniques have the potential to treat previously untreatable inherited genetic disorders of vision by delivery of normal copy of the gene of interest or correcting mutations that cause these afflictions. Although gene augmentation treatments are commercially available for inherited retinal diseases, there are many shortcomings that need to be addressed, like progressive retinal degeneration and diminishing efficacy over time. Innovative CRISPR-Cas9-based genome editing technologies have broadened the proportion of treatable genetic disorders and can greatly improve or complement treatment outcomes from gene augmentation. Progress in this relatively new field involves the development of therapeutics including gene disruption, ablate-and-replace strategies, and precision gene-correction techniques, such as base editing and prime editing. By making direct edits to endogenous DNA, genome editing theoretically guarantees permanent gene-correction and long-lasting treatment effects. Improvements to delivery modalities aimed at limiting persistent gene-editor activity have displayed an improved safety profile and minimal off-target editing. Continued progress to advance precise gene correction and associated delivery strategies will establish genome editing as the preferred treatment for genetic retinal disorders.
Please be advised that this is an in person event only and booking is mandatory as the seating is limited.
