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Nov. 15, 2006 — Recently there have
been some breakthroughs in our understanding of Parkinson's.
How might these findings affect the way we treat the
disease?
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Response
by Joshua Kritzer
Whitehead postdoctoral fellow |
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Much of what we know about Parkinson's disease comes
from two lines of inquiry. The first is human genetics,
studying how mutations in human genes cause familial,
inherited forms of Parkinson's disease. Many exciting
revelations have been made in this field in recent years,
including the identification of several genes that,
when they possess a specific mutation, appear to contribute
to familial Parkinson's. Although most Parkinson's is
not genetically inherited, but rather the "sporadic"
form of the disease, knowing which genes can contribute
to Parkinson's can help identify points in disease initiation
or progression that drugs could target. In other words,
discovering the genes behind familial Parkinson's points
the way to critical proteins whose functions could be
inhibited by drugs.
The second major line of inquiry into Parkinson's uses
models of the disease in other living systems. This
approach has also seen exciting developments lately.
By investigating how models of Parkinson's progress
in other living systems (including yeast, flies, worms,
mice and human cells in culture dishes) we are getting
a first glimpse into what goes wrong inside cells that
are dying in Parkinson's. If the same things that cause
yeast cells or worm neurons to go haywire in these models
also are behind neuronal dysfunction in Parkinson's,
then we can use these models to screen for drugs that
ameliorate the disease.
Together, these two approaches are revealing what is
actually going wrong on the cellular level in Parkinson's
disease. With that information, we are well-primed for
designing and testing new drugs and new therapies, which
with any luck will progress (in a matter of years to
decades) into clinical application.
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