(gentle music)
>> Huntington's disease is caused by death
of neurons in your brain.
The disorder involves
progressive loss of control
of movements, thoughts, and emotions.
It happens when a
mutation in a single gene
gets passed from a parent to a child.
Clinical trials of gene-based
therapies are beginning,
but these therapies must be given
directly into the central nervous system.
My name is Christopher Ross,
and my lab is studying a
complementary approach.
We've developed mouse models
which have progressive
symptoms similar to patients.
More recently, we've taken skin biopsies
directly from our patients
which can yield stem cells.
In the laboratory, we can
differentiate these stem cells
into neurons that die in
the same way as the disease.
We're studying chemical compounds
that can both improve
symptoms in the mouse models
and block cell death in the human neurons.
We're working with many other laboratories
throughout the world to
develop these compounds
into medications to delay the onset
or slow the progression of the condition.
I truly believe that working together
we can transform the treatment
of Huntington's disease.
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