Directed evolution of novel adeno-associated viral vectors for gene therapy

Published on August 5, 2014   52 min

Other Talks in the Series: Gene Transfer and Gene Therapy

0:00
This is David Schaffer. I'm a professor at the University of California at Berkeley in Chemical and Biomolecular Engineering, Bioengineering, and the Helen Wills Neuroscience Institute, as well as director of the Berkeley Stem Cell Center. In addition, I'm co-founder of the company, 4D Molecular Therapeutics. And I'm going to be talking about directed evolution of novel adeno-associated viral vectors for gene therapy.
0:23
So if we assess the current status of the field of gene therapy, which can be defined as the introduction or delivery of genetic material to the cells of an individual for therapeutic benefit. There have been a number-- an increasing number of successes over the past few years, and these include bona fide clinical successes for the treatment of Leber's congenital amaurosis, hemophilia B, as well as familial lipoprotein lipase deficiency and x-linked adrenoleukodystrophy. Now the first three indications I mentioned were made possible through adeno-associated viral vector technology, which has been increasingly successful as we mentioned over the past few years. Despite all of these successes however, a number of disease targets are still beyond the reach of current gene transfer technology. And that technology must be made better, which is the focus of my talk today.
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Directed evolution of novel adeno-associated viral vectors for gene therapy

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