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Microsatellite DNA Expansions in Rare Genetic Diseases

Microsatellite DNA Expansions in Rare Genetic Diseases

Tuesday, April 16, 2019

The New York Academy of Sciences, 7 World Trade Center, 250 Greenwich St Fl 40, New York

Presented By

Biochemical Pharmacology Discussion Group

The New York Academy of Sciences

 

Repeat expansion disorders encompass a heterogeneous group of more than 40 rare genetic diseases including Huntington disease, ALS/FTD, myotonic dystrophy, and spinocerebellar ataxias. While these neurodegenerative diseases are clinically diverse, they are all the result of the expansion of DNA microsatellite repeats, most frequently trinucleotide repeats. Although the sequences and length of repeats differ between diseases, common mechanisms underlie their pathology. Aberrant DNA repair and replication pathways are implicated as well as dysfunction in transcription and translation processes, and repeat-associated non-ATG translation. Given the severity of these diseases and the lack of effective therapeutics, there is an urgent need to improve our understanding of the mechanisms underlying the expansion of DNA microsatellite repeats. This event will explore recent developments in our knowledge of the biological processes driving repeat expansion diseases, as well as strategies for effective therapeutic intervention, and barriers to drug discovery.

Call for Abstracts

Abstract submissions are invited for a poster session and selected short talks. For complete submission instructions, please visit our online portal. The deadline for abstract submission is Monday, February 25,2019.

Registration

Member
By 03/04/2019
$90
After 03/04/2019
$130
Nonmember Academia, Faculty, etc.
By 03/04/2019
$180
After 03/04/2019
$260
Nonmember Corporate, Other
By 03/04/2019
$250
After 03/04/2019
$350
Nonmember Not for Profit
By 03/04/2019
$180
After 03/04/2019
$260
Nonmember Student, Undergrad, Grad, Fellow
By 03/04/2019
$100
After 03/04/2019
$145
Member Student, Post-Doc, Fellow
By 03/04/2019
$50
After 03/04/2019
$70
Earlybird Registration:
13
days
left
Deadline:
6
days
left

Organizers

Christine Bulawa, PhD,Pfizer
Christine Bulawa, PhD,
Pfizer
Sara Donnelly, PhD,The New York Academy of Sciences
Sara Donnelly, PhD,
The New York Academy of Sciences
Kari Fischer, PhD,The New York Academy of Sciences
Kari Fischer, PhD,
The New York Academy of Sciences
Aileen Healy, PhD,Pfizer
Aileen Healy, PhD,
Pfizer
Stefan McDonough, PhD,Pfizer
Stefan McDonough, PhD,
Pfizer
Laura Ranum, PhD,University of Florida
Laura Ranum, PhD,
University of Florida
Claire Steppan, PhD,Pfizer
Claire Steppan, PhD,
Pfizer

Keynote Speaker

Laura Ranum, PhD,University of Florida
Laura Ranum, PhD,
University of Florida

Speakers

Beverly Davidson, PhD,University of Pennsylvania
Beverly Davidson, PhD,
University of Pennsylvania
Yuanjing Liu, PhD
Yuanjing Liu, PhD

Wave Life Sciences

Joseph Nabhan, PhDPfizer
Joseph Nabhan, PhD
Pfizer
Christopher Pearson, PhD,The Hospital for Sick Children, University of Toronto
Christopher Pearson, PhD,
The Hospital for Sick Children, University of Toronto
Ekaterina Rogaeva, PhD,University of Toronto
Ekaterina Rogaeva, PhD,
University of Toronto
Peter Todd, MD, PhD,University of Michigan
Peter Todd, MD, PhD,
University of Michigan
Eric Wang, PhD,University of Florida
Eric Wang, PhD,
University of Florida
Vanessa Wheeler, PhD,Massachusetts General Hospital
Vanessa Wheeler, PhD,
Massachusetts General Hospital

Event Sponsors




Program Supporters




Tuesday

April 16, 2019

8:30 AM

Continental Breakfast and Registration

9:00 AM

Introduction and Welcome Remarks

Speakers

Kari Fischer, PhD
The New York Academy of Sciences
Christine Bulawa, PhD
Pfizer
9:15 AM

KEYNOTE ADDRESS: Repeat-Associated Non-ATG (RAN) Translation

Speaker

Laura Ranum, PhD
University of Florida

Session 1: Targeting the Repeats

10:00 AM

DNA Repair in Huntington’s Disease

Speaker

Vanessa Wheeler, PhD
Massachusetts General Hospital
10:30 AM

Networking Coffee Break

11:00 AM

Integrating Genetic and Epigenetic Data in C9orf72 Studies

Speaker

Ekaterina Rogaeva, PhD
University of Toronto
11:30 AM

Repeat-Associated Non-AUG (RAN) Initiated Translation in FXTAS Pathogenesis

Speaker

Peter Todd, MD, PhD
University of Michigan

Session 2: Data Blitz Talks

12:00 PM

2 X 5-minute Talks Selected from Submitted Abstracts

12:10 PM

Networking Lunch and Poster Viewing

Session 3: Co-opting Shared Pathological Consequences of Repeat Expansion for Therapeutic Approaches

1:10 PM

Optimization of RNAi Strategies for SCA1 and Huntington’s Disease

Speaker

Beverly Davidson, PhD
University of Pennsylvania
1:40 PM

Cas9 Therapy for Myotonic Dystrophy

Speaker

Eric Wang, PhD
University of Florida
2:10 PM

Disease Associated Repeat and Stability

Speaker

Christopher Pearson, PhD
The Hospital for Sick Children, University of Toronto
2:40 PM

Networking Coffee Break

Session 4: Challenges and Opportunities for Clinical Translation

3:10 PM

Paving the Pre-Clinical Path for Drug Development

Speaker

Joseph Nabhan, PhD
Pfizer
3:40 PM

Title To Be Announced

Speaker

Yuanjing Liu, PhD
Wave Life Sciences
4:10 PM

Panel Discussion: Barriers and Potential Solutions for Drug Development

4:55 PM

Closing Remarks and F1000 Poster Prize Presentation

5:10 PM

Networking Reception and Poster Viewing

6:10 PM

Adjourn