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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.
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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.
