Sigma-Aldrich receives award from The Michael J Fox Foundation to
develop clinical research models for Parkinson's disease
2 October 2009
Sigma-Aldrich (NASDAQ:SIAL) and The Michael J. Fox Foundation (MJFF)
have announced a collaboration that is expected to develop more accurate
preclinical research models of Parkinson’s disease, a neurodegenerative
disorder of unknown cause that affects nearly five million individuals
worldwide.
Under a research grant from MJFF, Sigma Advanced Genetic Engineering
(SAGE) Labs — an initiative of Sigma-Aldrich’s Research Biotech business
unit — will use novel CompoZr zinc finger nuclease (ZFN) technology in
an effort to create superior preclinical research models critically
needed for the development of transformative treatments for Parkinson’s
disease.
The models, which are expected to take as little as one year to
develop, will be made broadly accessible to scientists throughout the
Parkinson’s research community in order to speed basic research and drug
development efforts field-wide.
Though current mammalian models adequately recapitulate some outward
symptoms of Parkinson’s disease, no existing model has been able to
accurately mimic the onset and progression of the underlying disease
processes that characterize that disease in humans.
Research already conducted into the genetic causes of Parkinson’s
disease has identified a number of genes but indicates a strong
connection to mutations in five particular genes: LRRK2, alpha-synuclein,
DJ-1, Parkin and PINK1. MJFF funding will allow Sigma-Aldrich’s efforts
to create five novel rat models with each of these genes knocked out.
Adopting a new approach to developing more effective and targeted
research models, SAGE Labs will use the CompoZr ZFN technology in its
efforts to design ‘knockout’ rat models in which the genes known to be
directly implicated in Parkinson’s disease are omitted.
This research is expected to facilitate the development of new models
that scientists believe will provide a better understanding of
Parkinson’s disease at the molecular, biochemical, physiological and
behavioural levels. This knowledge may, in turn, result in new
therapeutic targets and approaches for the treatment of Parkinson’s
disease.
“Our Foundation’s mission is to accelerate the conversion of basic
discoveries about Parkinson’s disease into practical treatments that
will make a difference in patients’ lives,” said Katie Hood, CEO of The
Michael J. Fox Foundation. “To accomplish this, there is a critical need
for new research models to more effectively screen potential new
therapies, particularly neuroprotective therapies.
"We’re excited about the knockout rat technology developed by
Sigma-Aldrich, and hopeful for its prospects to quickly make a major
impact on Parkinson’s disease drug development efforts and help deliver
breakthrough treatments to patients faster.”
“The work undertaken and funding provided by The Michael J. Fox
Foundation is fundamental to the scientific community’s ability to get
one step closer to the next generation of Parkinson’s disease
treatments,” added Dr. Edward Weinstein, Director of SAGE Labs at
Sigma-Aldrich.
“With the power of our CompoZr technology, we are able provide a
unique and innovative contribution to furthering research into this
debilitating disorder. The models that we are developing may one day
help in the development of new therapeutic approaches to a number of
diseases. We are excited to have an opportunity to advance scientific
understanding in areas that can directly improve the lives of those that
suffer with this disease.”
Because rats are physiologically similar to humans, they are ideal
subjects for modelling human diseases and have been an important species
for research in a number of fields including physiology, endocrinology,
neurology, toxicology and cancer.
Until recently it has been impossible to create rat models with
particular genes deactivated, or ‘knocked out.’ However, using CompoZr
ZFN technology, scientists at SAGE Labs are able to generate animal
models with targeted genetic changes to better understand gene function
and develop new therapeutic approaches.
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