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Nanotechnology for intracellular nucleic acid delivery

Published on May 4, 2015   33 min

A selection of talks on Biochemistry

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0:00
It's a great pleasure for me to be one of the speakers for the Henry Stewart Talks. The title of my talk is, Nanotechnology for Intracellular Nucleic Acid Delivery.
0:12
Nucleic acids have become an indispensable research tool. And we can certainly say that. They have revolutionized biomedical research. From a pharmaceutical development point of view, they are at present mostly used for diagnostics and detection applications. Applications such as PCR, DNA microarrays and these bio-barcode assays can be used to accurately detect biomarkers that can be used to provide insights into disease processes and pinpoint pathways where treatments may interfere. What's perhaps even more exciting about nucleic acids in my opinion are their potential as therapeutics. One reason for this is that in principle, one change in disease target would only require a change in the nucleic acid sequence itself. So you can imagine that once a successful formulation of nucleic acid therapeutics is developed, subsequent formulations will follow much more easily. And this will likely bypass many of the steps involved in the development of a traditional small molecule drug. Because of the versatility and programmability of synthetic nucleic acids, there is a significant amount of hope and promise in developing nucleic acid therapeutics. And this has been manifested in an explosion of journal publications, journals dedicated to this study as well as the 2006 Nobel Prize awarded to Fire and Mello for their discovery of the RNA interference pathway. But surprisingly on the market, there are only a handful of nucleic acid therapeutics. And these include Macugen and Vitrovine. Both of these drugs must be directly injected at the site of pathology, which is in the vitreous of the eye. And the backbone of the nucleic acids be modified to enhance its stability. So this exemplifies the kind of difficulties associated with using nucleic acids as therapeutics, both regarding its physiochemical properties and biological properties.
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Nanotechnology for intracellular nucleic acid delivery

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