Genome Editing of Stem Cells: Moving Towards the Clinic
Genome editing provides a mechanism to precisely change the DNA sequence of cells with single nucleotide precision. We have developed a highly efficient platform to modify multiple types of human stem cells, including hematopoietic stem cells and pluripotent stem cells, with high efficiency using a combination of CRISPR/Cas9 and AAV6. I will discuss our progress in applying this platform to several different genetic diseases of the blood and immune system including sickle cell disease and severe combined immunodeficiency. In addition, I will discuss our optimized protocols to generate precisely modified human pluritpotent stem cells.
Date: 2 July 2018, 11:00
Venue: MRC Weatherall Institute of Molecular Medicine, Headington OX3 9DS
Venue Details: Seminar Room
Speaker: Dr. Matthew Porteus (Stanford University, School of Medicine)
Organising department: MRC Weatherall Institute of Molecular Medicine
Organiser: Sabrina Harris (Department of Paediatrics, University of Oxford)
Organiser contact email address: sabrina.harris@paediatrics.ox.ac.uk
Host: Georg Holländer (Department of Paediatrics, University of Oxford)
Part of: WIMM Occasional Seminars
Booking required?: Not required
Audience: Members of the University only
Editor: Sarah Butler