9:30 a.m. – 9:40 a.m. |
Welcome and Day 1 Introduction
Terry Watnick, M.D., Professor of Medicine, Joan B. and John H. Sadler Professor in Nephrology, and Principal Investigator, PKD RRC Coordinating Site, University of Maryland School of Medicine
Robert Star, M.D., Director, Division of Kidney, Urologic and Hematologic Diseases, National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH) |
9:40 a.m. – 10:00 a.m. |
The Patient Experience: Our Stories and Your Impact
Jullie Hoggan, PKD foundation volunteer and the facilitator of the Living Well with Kidney Disease Support group in Las Vegas |
10:00 a.m.- 10:05 a.m. |
Questions |
10:05 a.m. ‒ 11:20 a.m. |
Session 1: Ciliary Signaling |
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Session Chairs: Matthew Kavanaugh, M.D.- Ph.D., Physician Scientist Program, University of
Kansas Medical Center Gregory Pazour Ph.D., Professor, Program in Molecular Medicine, UMass Chan
Medical School
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10:05 a.m. – 10:25 a.m.
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Left, right and center: cilia and polycystin-2 as mechanotransducers during left-right axis development.
Shiaulou Yuan, Ph.D., Assistant Professor of Medicine, Harvard Medical School
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10:25 a.m. – 10:30 a.m. |
Questions |
10:30 a.m. – 10:50 a.m.
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ARL13B cilia localization and GEF activity in polycystic kidney disease
Robert E. Van Sciver, Ph.D., Postdoctoral Fellow, Emory University School of Medicine
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10:50 a.m. – 10:55 a.m. |
Questions |
10:55 a.m. – 11:00 a.m. |
Abstract Presentation
Role of Interferon-gamma in Cyst Formation After Acute Kidney Injury
Morgan E. Smith, University of Oklahoma Health Sciences Center
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11:00 a.m. – 11:05 a.m. |
Questions |
11:05 a.m. – 11:20 a.m. |
In Vivo Models subcommittee presentation and feedback session
Patricia Outeda Garcia, Ph.D., University of Maryland School of Medicine |
11:20 a.m. – 11:30 a.m. |
Break |
11:30 a.m. ‒ 1:15 p.m. |
Session 2: Is gene therapy for PKD on the horizon; Lessons learned from other inherited diseases. |
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Session Chairs: Vivek Bhalla, M.D., Associate Professor of Medicine, Stanford University Anubhav Chakraborty, Ph.D., Candidate, The University of Kansas Medical Center
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11:30 a.m. – 11:50 a.m. |
Learnings from neuromuscular disease gene therapies
Angela Lek, Ph.D., Vice President of Research, Muscular Dystrophy Association |
11:50 a.m. – 11:55 a.m. |
Questions |
11:55 a.m. – 12:15 p.m. |
The long and winding road to molecular cures for the first molecular disease.
John Tisdale, M.D., Branch Chief, Cellular and Molecular Therapeutics, NIH |
12:15 p.m. – 12:20 p.m. |
Questions |
12:20 p.m. – 12:40 p.m. |
Targeting the Kidneys with gene therapy
Matthew Wilson, M.D., Ph.D., Professor of Medicine, Vanderbilt University Medical Center |
12:40 p.m. – 12:45 p.m. |
Questions |
12:45 p.m. – 1:15 p.m. |
Lunch Break |
1:15 p.m. – 2:50 p.m. |
Session 3: Next steps in identifying PKD therapies: Brainstorming |
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Session Chairs: Neera Dahl, M.D., Ph.D., Senior Associate Consultant, Mayo Clinic
Stephen Seliger, M.D., Associate Professor, University of Maryland School of Medicine
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1:15 p.m. – 1:30 p.m. |
Therapy for PKD — Quo vadis?
Alan Yu, M.B., B. Chair., Professor of Medicine, The University of Kansas Medical Center |
1:30 p.m. – 1:35 p.m. |
Questions |
1:35 p.m. – 1:55 p.m. |
Brainstorming sessions – 4 breakout rooms (1 trainee session and 3 others)
Breakout Room Discussion Leaders:
Neera Dahl, M.D., Ph.D., Senior Associate Consultant, Mayo Clinic
Stephen Seliger, M.D., Associate Professor, University of Maryland School of Medicine
Vishal Patel, M.D., Professor, The University of Texas Southwestern Medical Center
Victoria Halperin-Kuhns, Ph.D., Research Associate, University of Maryland School of Medicine;
Amanda Clark, Ph.D., Postdoctoral Fellow, The University of Alabama at Birmingham; and Anubhav Chakraborty, Ph.D. Candidate, The University of Kansa Medical Center
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1:55 p.m. – 2:05 p.m. |
Break |
2:05 p.m. – 2:25 p.m. |
Summary of Breakout sessions – 4 breakout rooms |
2:25 p.m. – 2:40 p.m. |
Clinical subcommittee presentation feedback session
Stephen Seliger, M.D., Associate Professor, University of Maryland School of Medicine |
2:40 p.m. – 2:45 p.m. |
Break |
2:45 p.m. ‒ 4:05 p.m. |
Session 4: Tubuloids and Organoids |
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Session Chairs: Benjamin Freedman, Ph.D., Associate Professor, University of Washington
Isis Thomas, Ph.D. Candidate, Biomedical Science, The University of Alabama at Birmingham
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2:45 p.m. – 3:05 p.m. |
Using Stem Cells to Explore Genetics Underlying Brain Disease Kristen Brennand, Ph.D., Professor of Psychiatry, Yale University School of Medicine |
3:05 p.m. – 3:10 p.m. |
Questions |
3:10 p.m. – 3:30 p.m. |
Novel stem cell tools to model polycystic kidney disease Zhongwei Li, Ph.D., Assistant Professor, Keck School of Medicine, University of Southern California |
3:30 p.m. – 3:35 p.m. |
Questions |
3:35 p.m. – 3:40 p.m. |
Abstract Presentation
Deciphering the impact of RAC1-SPTAN1 in ARPKD Cystogenesis Using Multifaceted Models
Shohei Kuraoka, M.D., Ph.D., Postdoctoral Fellow Massachusetts General Hospital, Harvard Medical School |
3:40 p.m. – 3:45 p.m. |
Questions |
3:45 p.m. – 3:50 p.m. |
Abstract Presentation
Targeting ALDH1A1 Together With Nanoparticle Mediated Kidney Specific Immunotherapy Delays Cyst Growth in ADPKD Alice Shasha Cheng, Ph.D., Mayo Clinic |
3:50 p.m. – 3:55 p.m. |
Questions |
3:55 p.m. – 4:00 p.m. |
Abstract Presentation
O-GlcNAcylation, a Modulator of Metabolism and Ciliogenesis, Promotes Autosomal Dominant Polycystic Kidney Disease (ADPKD) Progression Matthew A. Kavanaugh, The University of Kansas Medical Center |
4:00 p.m. – 4:05 p.m. |
Questions |
4:05 p.m. |
Adjournment |