Surface-mediated targeting of lentiviral vectors

Published on August 5, 2014   29 min

Other Talks in the Series: Gene Transfer and Gene Therapy

0:00
This is Christian Buchholz at the Molecular Biotechnology and Gene Therapy section at the Paul-Ehrlich Institute in Langen, Germany. I'm very pleased to present the lecture on surface-mediated targeting of lentiviral vectors.
0:15
I have divided the lecture into the topics listed here. We'll start with an introduction into surface targeting of immuno viruses. I will then show you a series of examples covering a broad variety of target subtypes, including lymphocytes, hematopoietic stem cells, neurons, and endothelial cells. We will first focus on ex vivo applications and then go through some examples of in vivo administrations of the targeted vectors.
0:42
In gene therapy, we do basically distinguish between in vivo and ex vivo modification of target cells. By in vivo, we mean that vectors are directly injected into the patient, which can be locally, such as intracerebrally, or systemically. Ex vivo means that target cells are removed from the patient then transduced ex vivo in culture vessels before they will be re-implanted into the patient. It is obvious that upon in vivo applications, vectors will encounter a large variety of cell types besides the actual target cells relevant for therapy. However, also ex vivo, primary cells are not homogeneous and may well differ, for example, in the differentiation state. Targeting vector particles to the relevant cell type without losing particles to non-relevant cells is therefore an important goal in vector engineering. Only surface targeting of vector particles may make this possible.
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Surface-mediated targeting of lentiviral vectors

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