Gordon Research Conferences
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Conference Program
 
CAG Triplet Repeat Disorders
May 31 - June 5, 2009
Waterville Valley Resort
Waterville Valley, NH
Chair:
Jang-Ho Cha

Vice Chair:
Laura Ranum

Related Meeting Information
The CAG Triplet Repeat Disorders Gordon Research Conference was held in conjunction with the CAG Triplet Repeat Disorders Gordon-Kenan Research Seminar. Please refer to the CAG Triplet Repeat Disorders GKRS web page for more information.

The CAG Triplet Repeat Disorders are a group of largely untreatable inherited neurological disorders which result from an expansion in a CAG trinucleotide repeat in the mutant genes. This group of diseases includes Huntington’s disease, spinal and bulbar muscular atrophy, and several of the spinocerebellar ataxias. In each case, the CAG repeat lies within the coding region of a gene and results in an abnormally long polyglutamine tract within the mutant protein. Marked similarities in the underlying genetics and neuropathology suggest common pathologic mechanisms among these disorders. The 2009 Gordon Conference on CAG Triplet Repeat Disorders and the associated Gordon Research Seminar (GRS) will gather together young investigators and established senior scientists to deliver provoking lectures on the cutting-edge of science. Topics will include a clinical and pathological overview, circuitry malfunction, synaptic pathology, novel neuroimaging techniques, repeat instability, RNA gain of function, bidirectional expression, new animal models, and genetic modifiers. Strong emphasis is placed on training and mentoring of young scientists, and time will be devoted to career issues. All participants will be required to present posters. Some poster presenters will be selected for short talks. The collegial atmosphere of this Conference, with programmed discussion sessions as well as opportunities for informal gatherings in the afternoons and evenings, provides an avenue for scientists from different disciplines to brainstorm and promotes cross-disciplinary collaborations in the various research areas represented. Priority will be given to women, minorities and persons with disabilities when selecting participants.


Contributors
Cell Press
CHDI Foundation, Inc
Hereditary Disease Foundation
Kennedy’s Disease Foundation
National Ataxia Foundation
National Institutes of Health

SUNDAY
2:00 pm - 9:00 pmArrival and Check-in (Office Closed 6:00 pm - 7:00 pm)
6:00 pmDinner
7:30 pm - 7:40 pmWelcome / Introductory Comments by GRC Site Staff
7:40 pm - 9:30 pmKEYNOTE ADDRESS
Session Chair: Jang-Ho Cha (Massachusetts General Hospital)
7:40 pm - 8:40 pm Harry Orr (University of Minnesota)
"Biochemical and physiological deficits in a polyglutamine disease"
8:40 pm - 9:15 pm Discussion
MONDAY
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmCLINICAL PRESENTATION SETTING THE STAGE FOR PRECLINICAL OPPORTUNITIES (HD and SCAs)
Session Chair: Anne Young (Massachusetts General Hospital)
9:00 am - 9:30 am Marian DiFiglia (Massachusetts General Hospital)
"Molecular Pathology"
9:30 am - 9:45 am Discussion
9:45 am Coffee Break / Group Photo
10:30 am - 11:00 am Stefano DiDonato (Istituto Nazionale Neurologico 'C. Besta')
"Clinical Aspects and Peripheral Markers of Neurodegeneration"
11:00 am - 11:15 am Discussion
11:15 am - 11:45 am Jane Paulsen (University of Iowa)
"Biological and Clinical Markers of HD before Diagnosis: The PREDICT-HD Study"
11:45 am - 12:00 pm Discussion
12:00 pm - 12:30 pm Structured Discussion: Blair Leavitt (University of British Columbia)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session I
6:00 pmDinner
7:30 pm - 9:30 pmINFLAMMATION IN CAG TRIPLET REPEAT DISORDERS
Session Chair: Yijuang Chern (Institute of Biomedical Sciences, Taiwan)
7:30 pm - 8:00 pm Maria Bjorkqvist (Lund University)
"Peripheral markers of inflammation in HD"
8:00 pm - 8:15 pm Discussion
8:15 pm - 8:45 pm Thomas Moeller (University Washington)
"Role of Microglia in Huntington's disease"
8:45 pm - 9:00 pm Discussion
9:00 pm - 9:30 pm Structured Discussion: Paul Muchowski (University of California, San Francisco)
TUESDAY
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmTRANSCRIPTION: MECHANISMS AND THERAPY
Session Chair: Leslie Thompson (University of California, Irvine)
9:00 am - 9:30 am Ruth Luthi-Carter (Ecole Polytechnique Federale de Lausanne)
"Mechanisms of transcriptional dysregulation as revealed by gene expression profiling"
9:30 am - 9:45 am Discussion
9:45 am - 10:00 amPuneet Opal (Northwestern University)
"Role of transcriptional corepressors in SCA1 pathogenesis"
10:00 am Coffee Break
10:30 am - 11:00 am Gillian Bates (Kings College)
"HDAC inhibition in mouse models of HD"
11:00 am - 11:15 am Discussion
11:15 am - 11:45 am Joel Gottesfeld (Scripps Institute)
"Novel HDAC Inhibitors"
11:45 am - 12:00 pm Discussion
12:00 pm - 12:30 pm Structured Discussion: Joan Steffan (University of California, Irvine)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session II
6:00 pmDinner
7:30 pm - 9:30 pmGENETIC MODIFIERS
Session Chair: Diane Merry (Thomas Jefferson University)
7:30 pm - 8:00 pm Darren Monckton (University of Glasgow)
"Genetic modifiers of disease severity in myotonic dystrophy"
8:00 pm - 8:15 pm Discussion
8:15 pm - 8:45 pm Juan Botas (Baylor University School of Medicine)
"Genetic Modifiers in Drosophila"
8:45 pm - 9:00 pm Discussion
9:00 pm - 9:30 pm Structured Discussion: James Gusella (Massachusetts General Hospital)
WEDNESDAY
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmRNA GAIN OF FUNCTION AND BIDIRECTIONAL EXPRESSION
Session Chair: J. Paul Taylor (St Jude Children’s Hospital)
9:00 am - 9:30 am Tom Cooper (Baylor University School of Medicine)
"CUG effects on RNA splicing"
9:30 am - 9:45 am Discussion
9:45 am - 10:00 amPeng Jin (Emory University)
"Identification of small molecules suppressing rCGG-repeat-mediated neurodegeneration"
10:00 am Coffee Break
10:30 am - 11:00 am Al La Spada (University of Washington)
"Bidirectional expression in SCA7: role of CTCF repeats"
11:00 am - 11:15 am Discussion
11:15 am - 11:45 am Galina Filippova (Fred Hutchison Cancer Research Center)
"Bidirectional expression of DM1 and FMR1"
11:45 am - 12:00 pm Discussion
12:00 pm - 12:30 pm Structured Discussion: Maury Swanson (University of Florida)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session III
6:00 pmDinner
7:30 pm - 9:30 pmREPEAT INSTABILITY
Session Chair: Jenny Morton (Cambridge University)
7:30 pm - 8:00 pm Vanessa Wheeler (Massachusetts General Hospital)
"A genetics approach to understanding trinucleotide repeat instability in Huntington's disease"
8:00 pm - 8:15 pm Discussion
8:15 pm - 8:45 pm Christopher Pearson (Sick Kids Hospital)
"Tissue-specific determinants of trinucleotide repeat instability"
8:45 pm - 9:00 pm Discussion
9:00 pm - 9:30 pm Structured Discussion: Anne Messer (Wadsworth Institute)
THURSDAY
7:30 am - 8:30 amBreakfast
9:00 am - 10:00 amDATA BLITZ
Session Chair: Laura Ranum (University of Minnesota)
9:00 am - 9:10 am Heather Montie (GRS Attendee, Thomas Jefferson University)
"Cytoplasmic Retention of Polyglutamine-Expanded Androgen Receptor Ameliorates Disease via Autophagy in a Mouse Model of Spinal and Bulbar Muscular Atrophy"
9:10 am - 9:15 amDiscussion
9:15 am - 9:25 am Lise Munsie (GRS Attendee, McMaster University)
"The Huntington's Disease Protein has a Nuclear Function in Cell Stress Response"
9:25 am - 9:30 amDiscussion
9:30 am - 9:40 am Janghooo Lim (GRS Attendee, Baylor College of Medicine)
"Role of RNA regulation in SCA1 pathogenesis"
9:40 am - 9:45 amDiscussion
9:45 am - 9:55 am Scott Zeitlin (University of Virginia)
"Expression of a version of huntingtin lacking its short stretch of polyglutamine (deltaQ-htt) enhances neuronal autophagy and longevity in mice, and stimulates the formation of autophagosomes in vitro"
9:55 am - 10:00 amDiscussion
10:00 am Coffee Break
10:30 am - 12:30 pmNEUROIMAGING STUDIES
Discussion Leader: Yvette Bordelon (University of California, Los Angeles)
10:30 am - 11:00 am Gülin Öz (University of Minnesota)
"Magnetic Resonance Spectroscopy imaging studies of ataxia patients and mice"
11:00 am - 11:15 am Discussion
11:15 am - 11:45 am Wenzhen Duan (Johns Hopkins Univ)
"MRI as a biomarker in preclinical studies"
11:45 am - 12:00 pm Discussion
12:00 pm - 12:30 pm Structured Discussion: Yvette Bordelon (University of California, Los Angeles)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session IV
6:00 pmDinner
7:00 pm - 7:30 pmBusiness Meeting
(Nominations for the next Vice Chair; Fill out Conference Evaluation Forms; Discuss future Site & Scheduling preferences; Election of the next Vice Chair)
7:30 pm - 9:30 pmCIRCUITRY MALFUNCTION AND SYNAPTIC PATHOLOGY
Session Chair: Lynn Raymond (Univ British Columbia)
7:30 pm - 8:00 pm Xiao-Jiang Li (Emory University)
"Synaptic pathology in CAG Triplet Repeat Disorders"
8:00 pm - 8:15 pm Discussion
8:15 pm - 8:45 pm George Rebec (Indiana University)
"A systems-level analysis of HD mouse models: dysregulation of ascorbate release, glutamate uptake, and corticostriatal neuronal communication"
8:45 pm - 9:00 pm Discussion
9:00 pm - 9:30 pm Structured Discussion: Kerry Murphy (Open University)
FRIDAY
7:30 am - 8:30 amBreakfast
9:00 amDepart

Funding for this conference was made possible (in part) by 1R13NS065503 from the National institute of Neurological Disorders and Stroke (NINDS) and Office of The Director, National Institutes of Health (OD). The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.

 
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