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Conference Program
 
CAG Triplet Repeat Disorders
Basic Science to Therapeutics
June 23-28, 2013
Waterville Valley Resort
Waterville Valley, NH
Chair:
Leslie M. Thompson

Vice Chair:
Diane E. Merry

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

Trinucleotide CAG expansion mutations cause a number of progressive neurodegenerative disorders including Huntington’s disease (HD), the spinocerebellar ataxias (SCAs) 1, 2, 3, 6, 7 and 17, and spinal and bulbar muscular atrophy (SBMA). These diseases are thought to primarily result from aberrant protein gain-of-function effects, although the recent discovery of antisense transcripts at several of these loci and a new appreciation for the complexity of gene expression and epigenetic changes across the genome has raised additional possibilities. The 2013 Gordon Conference on CAG Triplet Repeat Disorders and the associated Gordon Research Seminar (GRS) will bring together scientists from around the world to discuss cutting-edge research on the molecular and cellular mechanisms of these disorders and the various therapeutic strategies which are being developed to combat them. Meeting topics will include: early clinical signs and biomarkers, proteastasis, protein structure, gene and environment interactions, inflammation and peripheral immune system biology, cell metabolism, defining endophenotypes and associated disease mechanisms, interneuron interactions, synaptic plasticity, RNA gain-of-function, stem cell biology and therapeutic strategies. Strong emphasis will be placed on training and mentoring of young scientists with time devoted to career issues. All participants will be required to present posters and some poster presenters will be selected for short talks. The collegial atmosphere of this Conference, with structured discussion sessions and opportunities for informal afternoon and evening gatherings will provide an excellent opportunity for scientists from different disciplines to brainstorm with each other and to establish interdisciplinary collaborations.


Contributors

SUNDAY
2:00 pm - 9:00 pmArrival and Check-in
6:00 pmDinner
7:30 pm - 7:40 pmWelcome / Introductory Comments by GRC Site Staff
7:40 pm - 9:30 pmClinical Signs, Biomarkers and Systemic Effects
Chair: Erich Wanker (Max Delbruck Center for Molecular Medicine, Germany)
7:40 pm - 8:15 pmAnne B. Young (Mass General Hospital, Boston, USA)
"Perspectives on clinical trials and biomarkers"
8:15 pm - 8:25 pmDiscussion
8:25 pm - 9:00 pmKenneth Fischbeck (National Institutes of Health, USA)
"Historical and therapeutic insights into CAG repeat disease"
9:00 pm - 9:10 pmDiscussion
9:10 pm - 9:30 pmStructured Discussion
Discussion Leader: Beverly Davidson (University of Iowa, USA)
MONDAY
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmProteostasis
Chair: Eric Reits (University of Amsterdam, Netherlands)
9:00 am - 9:30 amSteven Finkbeiner (Gladstone/University of California, San Francisco, USA)
"Proteostasis in HD"
9:30 am - 9:45 amDiscussion
9:45 am - 10:15 amJason Gestwicki (University of Michigan, USA)
"Using the Hsp70/Hsp90 Chaperone System for the Treatment of Polyglutamine Expansion Disorders"
10:15 am - 10:30 amDiscussion
10:30 am - 11:00 amGroup Photo / Coffee Break
11:00 am - 11:10 amJoan S. Steffan (University of California, Irvine, USA)
"Is Huntingtin a scaffold for selective autophagy?"
11:10 am - 11:15 amDiscussion
11:15 am - 11:45 amJudith Frydman (Stanford University, USA)
"Suppression of Htt aggregation by the chaperonin TRIC"
11:45 am - 12:00 pmDiscussion
12:00 pm - 12:10 pmNicholas Caron (McMaster University, Canada)
"The Importance of Flexible Polyglutamine Tracts in CAG Trinucleotide Disorders"
12:10 pm - 12:15 pmDiscussion
12:15 pm - 12:30 pmStructured Discussion
Discussion Leader: Ai Yamamoto (Columbia University, USA)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
6:00 pmDinner
7:30 pm - 9:30 pmMolecular and Cellular Sequelae of Polyglutamine Expansion
Chair: Janet Dubinsky (University of Minnesota, USA)
7:30 pm - 8:00 pmErnest Fraenkel (Massachusetts Institute of Technology, USA)
"Effects of mutant huntingtin on DNA methylation"
8:00 pm - 8:10 pmDiscussion
8:10 pm - 8:40 pmChristopher Gomez (University of Chicago, USA)
"SCA6: expanded CAG repeats disrupt transcription factor function of a calcium channel gene"
8:40 pm - 8:50 pmDiscussion
8:50 pm - 9:20 pmJ. Lawrence Marsh (University of California, Irvine, USA)
"Fly models of HTT cleavage products"
9:20 pm - 9:30 pmDiscussion
TUESDAY
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmCell Metabolism
Chair: Kimberly Kegel (Mass General Institute for Neurodegenerative Diseases)
9:00 am - 9:30 amJeffrey Carroll (Western Washington University, USA)
"Altered flux from glucose in cells and tissues from HD knock-in mice"
9:30 am - 9:45 amDiscussion
9:45 am - 10:15 amAndrew Dillin (Salk Institute, USA)
"A novel mitochondrial response to expanded polyglutamine proteins"
10:15 am - 10:30 amDiscussion
10:30 am - 11:00 amCoffee Break
11:00 am - 11:10 amAudrey Dickey (University of California, San Diego, USA)
"Impaired PPAR delta function underlies mitochondrial demise and neurodegeneration in Huntington's disease: mechanistic basis and therapeutic implications"
11:10 am - 11:15 amDiscussion
11:15 am - 11:45 amCynthia T. McMurray (Lawrence Berkeley Laboratory, USA)
"Caveolar-related cholesterol trafficking and metabolic contributions to HD toxicity in vitro and in vivo"
11:45 am - 12:00 pmDiscussion
12:00 pm - 12:10 pmJohn Lee (University of Iowa, USA)
"Rhes modulation reveals impaired mTORC1 activity as a key contributor to striatal atrophy and metabolic deficits in Huntington's disease"
12:10 pm - 12:15 pmDiscussion
12:15 pm - 12:30 pmStructured Discussion
Discussion Leader: Al La Spada (University of California, San Diego, USA)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
6:00 pmDinner
7:30 pm - 9:30 pmEmerging Insights
Chair: Dimitri Krainc (Mass General Hospital, USA)
7:30 pm - 8:00 pmChristopher E. Pearson (The Hospital for Sick Children, Canada)
"MSH3 polymorphisms and protein levels affect CAG repeat instability in HD mice"
8:00 pm - 8:15 pmDiscussion
8:15 pm - 8:45 pmLaura Ranum (University of Florida, USA)
"Repeat Associated Non-AUG Translation in Microsatellite Expansion Disorders"
8:45 pm - 9:00 pmDiscussion
9:00 pm - 9:10 pmAndreas Neueder (King's College, UK)
"Aberrant splicing of HTT generates pathogenic exon 1 protein in Huntington disease"
9:10 pm - 9:15 pmDiscussion
9:15 pm - 9:30 pmStructured Discussion
Discussion Leader: Peter Todd (University of Michigan, USA)
WEDNESDAY
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmInflammation/Peripheral immune biology
Chair: Aleksey Kazantsev (Massachusett General Hospital, USA)
9:00 am - 9:30 amAsa Petersén (Lund University, Sweden)
"Hypothalamic changes and metabolic dysfunction in Huntington's disease"
9:30 am - 9:45 amDiscussion
9:45 am - 10:15 amElaine Hsiao (Caltech, USA)
"Learning to Tolerate our Microbial Self"
10:15 am - 10:30 amDiscussion
10:30 am - 11:00 amCoffee Break
11:00 am - 11:10 amAnnie Sittler (Université Pierre et Marie Curie-Paris, France)
"Interferon-beta induces clearance of mutant ataxin-7 and improves locomotion in SCA7 knock-in mice"
11:10 am - 11:15 amDiscussion
11:15 am - 11:45 amFlaviano Giorgini (University of Leicester, UK)
"KMO as a therapeutic target in Huntington's disease: recent advances"
11:45 am - 12:00 pmDiscussion
12:00 pm - 12:10 pmAndrew Lieberman (University of Michigan Medical School, USA)
"Peripheral androgen receptor gene silencing rescues disease in SBMA mice"
12:10 pm - 12:15 pmDiscussion
12:15 pm - 12:30 pmStructured Discussion
Discussion Leader: Sarah Tabrizi (University College London, UK)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
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 pmEndophenotypes and associated disease mechanisms
Chair: Scott Zeitlin (University of Virginia, USA)
7:30 pm - 8:00 pmAnthony Hannan (Florey Institute, Australia)
"Gene-environment interactions modulating cognitive and affective endophenotypes in Huntington's disease transgenic mice"
8:00 pm - 8:15 pmDiscussion
8:15 pm - 8:25 pmKarolina Kolodziejczyk (University of British Columbia, Canada)
"The differential effect of thalamic vs. cortical input onto striatal medium sized spiny neurons in the YAC128 mouse model of Huntington's disease in novel coculture systems"
8:25 pm - 8:30 pmDiscussion
8:30 pm - 9:00 pmMichael S. Levine (University of California, Los Angeles, USA)
"Interneurons in Striatum and Cortex in HD"
9:00 pm - 9:15 pmDiscussion
9:05 pm - 9:30 pmStructured Discussion
Discussion Leader: Tim Ebner (University of Minnesota, USA)
THURSDAY
7:30 am - 8:30 amBreakfast
9:00 am - 12:30 pmTherapeutics
Chair: Ruth Luthi-Carter (University of Leicester, UK)
9:00 am - 9:30 amClive Svendsen (Cedars Sinai, USA)
"Modeling repeat disorders using induced pluripotent stem cells: Lessons from HD and ALS"
9:30 am - 9:45 amDiscussion
9:45 am - 10:15 amBenjamin Shapiro (University of Maryland at College Park, US)
"Manipulating Magnetic Fields to Direct Nanotherapeutics to Ears, Eyes, and the Brain"
10:15 am - 10:30 amDiscussion
10:30 am - 11:00 amCoffee Break
11:00 am - 11:10 amChristian Neri (INSERM, France)
"Showcasing the capability of the Biogemix platform for cross-species gene prioritization in Huntington's disease"
11:10 am - 11:15 amDiscussion
11:15 am - 11:45 amBernhard Landwehrmeyer (University of Ulm, Germany and CHDI Foundation, USA)
"Overview of Therapeutic Landscape for HD"
11:45 am - 12:00 pmDiscussion
12:00 pm - 12:10 pmEdward Wild (UCL Institute of Neurology, UK)
"Knowledge is power: communication in neuroscience"
12:10 pm - 12:15 pmDiscussion
12:15 pm - 12:30 pmStructured Discussion
Discussion Leader: Neil Aronin (University of Massachusetts Medical School, USA)
12:30 pmLunch
1:30 pm - 4:00 pmFree Time
4:00 pm - 6:00 pmPoster Session
6:00 pmDinner
7:30 pm - 9:30 pmKeynote Session: Moving Forward in Treatment
Chair: Blair Leavitt (University of British Columbia)
7:30 pm - 8:00 pmJang-Ho Cha (Merck)
"Therapeutics in neurodegeneration"
8:00 pm - 8:15 pmDiscussion
8:15 pm - 9:15 pmKeynote Talk: Gen Sobue (Nagoya University, Japan)
"Molecular targeted therapy for spinal and bulbar muscular atrophy"
9:10 pm - 9:30 pmDiscussion
FRIDAY
7:30 am - 8:30 amBreakfast
9:00 amDeparture

Research reported in this publication was supported by the National Institute Of Neurological Disorders And Stroke of the National Institutes of Health under Award Number R13NS083250. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

 
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