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J Neurol Neurorehabil Res 2017 | Volume 2 Issue 3
allied
academies
17
th
International Conference on
4
th
International Conference on
NEUROLOGY AND NEUROSCIENCE
&
MENTAL HEALTH AND PRIMARY CARE
October 16-18, 2017 | Toronto, Canada
Wenzhen Duan
Johns Hopkins University, USA
Therapeutic target and biomarker development in Huntington’s disease
M
ost neurodegenerative diseases, including Alzheimer’s,
Parkinson’s, and Huntington’s disease (HD), have
convergingpathogenesis, suchas formationof abnormal protein
aggregates and mitochondrial dysfunction in the nervous
system. Unfortunately, despite tremendous efforts by many
scientists and increasing knowledge about diseasemechanisms,
we still lack disease-modifying treatments for any of these
diseases. While these diseases affect different areas of the
brain and are distinct at the cellular and molecular levels, they
share underlying similarities. Thus, development of treatment
for any one disease has the potential to accelerate the path to
treatment for related neurodegenerative diseases. Research
into potential therapies for HD is particularly attractive because
it is a genetically homogeneous disease for which numerous
well-established animal and cell-based models exist. HD is an
autosomal dominant disease caused by a CAG repeat expansion
inexon1ofthehuntingtingene.TheHDgeneencodestheprotein
huntingtin (Htt), whose polyglutamine expansion is believed
to mediate the cytotoxic effects of HD. Therefore, HD serves a
model for both neurodegenerative diseases and polyglutamine
diseases. Our laboratory aims to develop therapeutic targets
and biomarkers for neurodegenerative diseases, with a focus on
HD. I will discuss our recently identified therapeutic targets as
well as biomarkers which have high potentials to be translated
into clinical application. Drug discovery has been revolutionized
in the past decade. However, despite technological advances
because of substantial investment, the number of new drug
approvals remains stagnant and the cost of bringing a drug to
market is higher than ever. This highlights the persistence of a
model of drug development that has not adapted to changes
in science and public perception of drug companies. I will use
Huntington’s disease as an example to discuss the challenges
and opportunities in the translational neurobiology and drug
development.
Speaker Biography
Wenzhen Duan is an Associate Professor of Psychiatry and Neuroscience, Johns Hopkins
University School of Medicine, has completed her PhD in Neuropharmacology at Peking
Union Medical University, China in 1998. She is the current Director of Laboratory of
Translational Neurobiology at Johns Hopkins University School of Medicine. She is
internationally known for her work on translational research in neurodegenerative
diseases, particularly in Huntington’s disease. She is a pioneer in developing multimodal
micro-MRI biomarkers for preclinical studies of neuroprotective therapeutics. Her
laboratory identified key molecular targets for therapeutic intervention for HD and
conducted preclinical therapeutic trials. She has published over 70 research articles
and reviews. She is recognized by national and international organizations and serves
in Review Committee for science foundations in the USA such as NIH as well as outside
US, including UK, Austria, Swiss, Italian, and other European science foundations.
e:
wduan2@jhmi.edu