Gene transfer strategies: principles, state-of-the-art and the major barriers that need to be overcome 1

Published on August 5, 2014   47 min

Other Talks in the Category: Diseases, Disorders & Treatments

0:00
So hello, everybody. This so the first recording in this series of gene therapy. I am in the Luigi Naldini. I am the director of the San Raffaele-Telethon Institute for Gene Therapy in Milan, Italy. And I will be discussing, in this first lecture, general features of gene therapy and vectors.
0:22
In the first slide, we are mentioning the key strategies in gene transfer and gene therapy. Gene addition, which is the insertion of new genetic information in a cell to replace a malfunctioning gene and replacing its function, or to instruct a novel function to that cell-- for instance, to make that cell resistant to an infectious agent or to help that cell better fight the cancer. These are the most common strategies in gene transfer and therapy today. There are, however, additional strategies, which are gene subtraction-- in which case, we aim to disrupt or inhibit an endogenous gene to essentially prevent its expression, again, to enable maybe resistance to a virus or to fight a dominant oncogenic mutation. And gene editing, which is a more advanced of gene therapy in which, rather than adding or subtracting genes, we are actually rewriting the genetic sequencing in the cell, most often to correct directly in situ, a genetic inherited mutation, or to insert novel sequence at this specific site. And gene editing can be performed at the RNA level, in which case we would modify RNA transcript, either by, for instance, forcing axon skipping or trans-splicing. Or in a more stable at the DNA level by either disrupting genes or forcing homologous recombination through the use of artificial nucleuses. And all of these strategies will be, of course, discussed in detail in selected talks.
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Gene transfer strategies: principles, state-of-the-art and the major barriers that need to be overcome 1

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