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Gene therapy and gene transfer: vector integration preferences and integration site analysis

Published on August 5, 2014   50 min

A selection of talks on Infectious Diseases

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0:00
My name is Frederic Bushman. I'm a professor at the University of Pennsylvania. And the title of this lecture is Vector Integration Preferences and Integration Site Analysis.
0:12
This lecture will begin with an introduction, just a little bit about gene therapy and vector integration. Then methods for monitoring gene transfer. Then HIV integration targeting. Then gammaretroviral integration targeting, which differs. And lastly, analysis of vector integration in human gene therapy.
0:35
Next slide shows the general idea of stem cell gene therapy to treat an inherited disease. So bone marrow cells are removed from the patient, and then gene-corrected by transduction ex vivo with retroviral vector that encodes the corrective gene, shown in the upper left. So that installs the corrective gene into bone marrow cells, lower left, where each cell now has the vector integrated at a different location in the human genome. You can then re-infuse those cells into the subject. And bone marrow cells produce daughter cells, which circulate in the blood. You can then sample cells from blood years later, shown on the right, and sequence integration site distributions. And that can tell you things like how many different kinds of cells; how many different progenitor cells or contributing cells to the periphery; and whether some cells are contributing more than others; and in cases of adverse events, leukemia, you can tell where the integration site was and get some information on the genes involved. So stem cell gene therapy has been used to treat many subjects now with inherited immuno-deficiencies and other conditions.

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