Combinatorial Vector Engineering for Gene Therapy Research

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Nov 15, 2013 (3 years and 10 months ago)

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The Gene and Linda Voiland School of Chemical
Engineering and Bioengineering

Seminar

Monday, March 9, 2009

4:10 p.m.

ETRL 101








Combinatorial Vector Engineering for Gene Therapy
Research

presented by Dr. Aresh Hatefi

Assistant Professor Pharmaceutical Sciences

Washington State University


In recent years, scientific interest in nature, particularly in the fields of
engineering and life sciences, has grown tremendously. The science of
biomimetics has the potential to advance many areas of technology including
vector development for gene therapy research. Biomimetic vectors are designed
to mimic the viral characteristics and perform a collection of self
-
guided
functions in order to overcome the cellular barriers. For administered
therapeutic genes to successfully reach the nucleus of the target cells, a carrier
(vector) should be designed to overcome various cellular barriers. Accordingly,
the carrier should be able to: a) condense DNA from a large micro
-
meter scale to
a smaller nano
-
meter scale suitable for endocytic uptake and protection from
nuclease degradation, b) be recognized by specific receptors on the target cells
and internalize, c) promote the escape of the gene from the endosomal
compartment into the cytosol, and d) assist the translocation of DNA from the
cytosol to the nucleus.


The overall objective of the research in our lab is to develop a gene delivery
system that is customizable, easy to engineer, efficient and non
-
toxic. As a first
step towards achieving the objective, a series of multifunctional biomimetic
vectors were genetically engineered and characterized to identify the structure
of the most suitable construct for targeted gene transfer.