Advanced genetic engineering of hematopoiesis: how to select the best engineering tool and expand target diseases and scope of intervention
Recording not available
Speaker: Prof. Luigi Naldini, Professor of Cell and Tissue Biology and of Gene and Cell Therapy at the San Raffaele University School of Medicine and Scientific Director of the San Raffaele Telethon Institute for Gene Therapy, Milan, Italy.
When aiming to genetically engineer hematopoietic stem cells, translational scientists face a constantly expanding toolbox where every newly added tool promises more precise and safer performance. This talk will address how to guide the choice of the best fitting strategy for a growing range of applications among capitalizing on older tools, such as lentiviral vector mediated gene replacement, which have an established track record of successful clinical use but lack precision, or capturing the promise of targeted gene editing by artificial nucleases, which is showing preliminary but encouraging success in the clinic but still faces outstanding challenges and unknowns in the long-term, or even to embrace the newest kids in the block and venturing into the realm of break-free editors.
