Gordon Research Conferences
Meeting Details

CAG Triplet Repeat Disorders (GRS)
Gordon Research Seminar

CAG Repeat Expansion Diseases: From Basic Mechanisms to Translational Science


June 3-4, 2017


Mount Snow
West Dover, VT


Andreas Neueder & Colleen A. Stoyas

Meeting Description

The Gordon Research Seminar on CAG Triplet Repeat Disorders is a unique forum for graduate students, post-docs, and other scientists with comparable levels of experience and education to present and exchange new data and cutting edge ideas.

The focus of the this GRS is to bring together young scientists from around the world to present and discuss their recent findings on the mechanisms of pathology underlying CAG triplet repeat disorders, as well as on advancements in how to combat these diseases. The meeting will feature a keynote presentation, data presentations from young scientists, and poster presentations. Throughout the meeting, attendees will have the opportunity to engage with peers and discuss different career options while networking with mentors from academia and industry.

Related Meeting

This GRS was held in conjunction with the "CAG Triplet Repeat Disorders" Gordon Research Conference (GRC). Refer to the associated GRC program page for more information.


Meeting Program

2:00 pm - 5:00 pmArrival and Check-in
3:30 pm - 3:45 pmIntroductory Comments by GRC Site Staff / Welcome by the GRS Conference Chair
3:45 pm - 4:30 pmKeynote Session: A Scientific Journey: Passion, Flexibility and Collaboration
Discussion Leader: Philipp Trepte (Max Delbrueck Center for Molecular Medicine in the Helmholtz Association, Germany)
3:45 pm - 4:20 pmLeslie Thompson (University of California, Irvine, USA)
"A Scientific Journey: Passion, Flexibility and Collaboration"
4:20 pm - 4:30 pmDiscussion
4:30 pm - 6:00 pmPoster Session
6:00 pmDinner
7:30 pm - 9:30 pmEmerging Methods and Models: Illuminating New Paths to Therapeutics
Discussion Leader: Shyam Ramachandran (Children's Hospital of Philadelphia, USA)
7:30 pm - 7:50 pmAlexander Tereshchenko (University of Iowa, USA)
"The Role of the Cerebellum in Juvenile Huntington's Disease (JHD)"
7:50 pm - 8:00 pmDiscussion
8:00 pm - 8:20 pmLauren Byrne (UCL Huntington's Disease Centre, United Kingdom)
"Towards a Readily Accessible Biofluid Biomarker for Huntington's Disease"
8:20 pm - 8:30 pmDiscussion
8:30 pm - 8:50 pmPamela Farshim (University College London, United Kingdom)
"Systemic Administration of a Novel AAV Variant Results in Widespread and Efficient Gene Transfer in R6/2 Mice"
8:50 pm - 9:00 pmDiscussion
9:00 pm - 9:20 pmDaniel Child (The Children's Hospital of Philadelphia, USA)
"Abnormal Cardiac mTORC1 Activity in Huntington Disease Mouse Models"
9:20 pm - 9:30 pmDiscussion
7:30 am - 8:30 amBreakfast
9:00 am - 11:00 amMolecular Mechanisms of Disease
Discussion Leader: Gunasekhar Burra (Indian Institute of Technology Kanpur, India)
9:00 am - 9:20 amAnne Steinhof (Max Delbrueck Center for Molecular Medicine in the Helmholtz Association, Germany)
"Mutant HTT Seeding Activity: A Marker of Disease Progression in Models of Huntington's Disease"
9:20 am - 9:30 amDiscussion
9:30 am - 9:50 amSamir Nath (University of Michigan, USA)
"Proteasome Dysregulation in Spinobulbar Muscular Atrophy"
9:50 am - 10:00 amDiscussion
10:00 am - 10:20 amCharlene Geater (University of California, Irvine, USA)
"Huntington's Disease in a Dish"
10:20 am - 10:30 amDiscussion
10:30 am - 10:50 amRadhia Kacher (CNRS / Pierre and Marie Curie University, France)
"zQ175 Knock-In Mouse Model Helps to Decipher the Neuroprotective Role of CYP46A1, the Cholesterol Degradation Enzyme, in Huntington's Disease"
10:50 am - 11:00 amDiscussion
11:00 am - 12:30 pmPoster Session

Coffee will be served in the poster area from 11:00 am - 11:30 am
12:30 pmLunch
1:30 pm - 2:30 pmPathophysiology of Neurodegeneration
Discussion Leader: Sarah Gardiner (Leiden University Medical Centre, The Netherlands)
1:30 pm - 1:45 pmFrederick Arnold (Thomas Jefferson University, USA)
"Deficient Nuclear Export of Polyglutamine-Expanded Androgen Receptor Contributes to Toxicity in a Cell Model of Spinal and Bulbar Muscular Atrophy"
1:45 pm - 1:50 pmDiscussion
1:50 pm - 2:05 pmAnastasia Gromova (University of California, San Diego, USA)
"Elucidating Mechanisms of Skeletal Muscle-Driven Neurodegeneration in Spinal and Bulbar Muscular Atrophy"
2:05 pm - 2:10 pmDiscussion
2:10 pm - 2:25 pmNikhil Ratna (National Institute of Mental Health and Neurosciences, India)
"Lithium in Huntington's Disease"
2:25 pm - 2:30 pmDiscussion
2:30 pm - 3:00 pmEvaluation Period
Fill in GRS Evaluation Forms
3:00 pmSeminar Concludes

Funding for this conference was made possible (in part) by 1R13NS103418-01 from the National Institutes of Health. 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 of trade names, commercial practices, or organizations imply endorsement by the U.S. Government.
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