PRF 11th Anniversary Workshop on Progeria - "Hand in Hand: Basic and Clinica

Progeria Research Foundation, Inc., Peabody MA

Investigators

Abstract

DESCRIPTION (provided by applicant): Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) is a rare, fatal segmental premature aging disease caused by a splice site mutation in LMNA. All children with Progeria die from heart attacks or strokes at an average age of 13 years following premature, progressive atherosclerosis. The mission of The Progeria Research Foundation (PRF) is to find the cause, treatments and cure for Progeria and its aging-related disorders. The 2013 PRF International Scientific Workshop on Progeria will be held in Bethesda, MD on April 24-26, 2013. This will be the sixth in a perpetual series conducted by PRF since 2001. All prior meetings were co-funded by the National Institutes of Health. With record-high peer-reviewed publications on Progeria in 2011 and 2012, there is a tremendous amount of new scientific information to discuss. Meeting Objectives: to create an ideal environment for collaborative discussion between basic and clinical scientists about how their collective experience with Progeria can accelerate the scientific progress in the fields of Progeria, cardiovascular disease (CVD) and aging. Program and Speakers: The meeting starts with an inspiring interview session with children living with Progeria. This is followed by Plenary Session speaker Francis Collins, MD, PhD, who discovered the Progeria gene mutation. He will focus on Progeria as a translational medicine success story which brings us unique keys to understanding elements of cardiovascular disease and aging. In a session entitled Clinical Treatment Trials, trial principal investigators will present major findings. Mark Kieran, MD, PhD (Dana Farber Cancer Inst., Boston) and Nicolas Levy, MD (Marseilles, France) will discuss farnesyltransferase inhibitor, bisphosphonate and statin use in Progeria. Clinical experts on CVD and bone disease of Progeria and aging will also present. George Martin, MD (U. Washington), a top expert in the genetics of aging, will lead the session entitled Aging, Cardiovascular Disease, and Progeria, which will include investigations on the connections between progerin and telomeres. Carlos L¿pez-Ot¿n, PhD (Oviedo, Spain) will moderate a session on progerin and lamin partners in health and disease, with leading lamin biologists such as Robert Goldman, PhD (Northwestern U.). Thomas Misteli, PhD (NCI) will lead a session on cutting edge strategies for research and discovery, including induced pluripotent stem cell research, genetic therapies, rapamycin and drug discovery. Two short-talk panel sessions will foster young investigators. Each talk will be accompanied by a poster with study details. Judith Campisi, PhD (Buck Inst.), a world-renowned aging investigator will moderate a basic science panel; Monica Kleinman, MD (Boston Children's Hosp.), who has led clinical policy panels for the American Heart Association and co-chairs Progeria clinical trials, will moderate a clinical science panel. We estimate 155 attendees, 39 speakers and 50 poster presentations. Educational tools include a poster competition and continuing medical education credits. Conclusion: Science presented at this meeting represents the next wave of discovery in Progeria and its relationships to CVD and aging.