Overview: Hutchinson-Gilford progeria syndrome (HGPS or Progeria) is a rare, fatal segmental premature aging disease caused by a splice site mutation in the LMNA gene. Children with Progeria die from heart attacks or strokes at an average age of 14.6 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, including heart disease. The upcoming PRF International Scientific Workshop on Progeria will be held in Cambridge, MA on September 20-22, 2018. This will be the 9th in a perpetual series of International Workshops conducted by PRF since 2001. All prior meetings were co-funded by the National Institutes of Health. With record-high numbers of both peer-reviewed publications on Progeria and requests for cell lines from the PRF Cell and Tissue Bank for HGPS preclinical explorations, and the recent launch of an international clinical treatment trial, there is a tremendous amount of new scientific information to discuss. Objectives: to create an ideal environment for collaborative discussion between basic and clinical scientists about how their collective experience with Progeria can accelerate scientific progress in the fields of Progeria, cardiovascular disease (CVD) and aging; to coordinate research activities, and to develop future activities in these fields. Program: The meeting starts with an inspiring evening session where children living with Progeria, along with their parents, share their experiences, feelings, and extraordinary personalities with the scientific audience. This is followed by a plenary presentation by Eric S. Lander, PhD (Broad Inst.), who will apply his vast experience with emerging scientific and technologic advances to the field of Progeria research as it relates to CVD, aging, and the discovery of new treatments. Day two will start with a comprehensive ?baseline? session summarizing key clinical and basic science discoveries in HGPS and their relationships to aging and CVD. These opening discussions will lay the foundation for more in-depth exploration of specific aspects of HPGS in the subsequent sessions. Peppered throughout the program are seasoned HGPS, CVD and aging experts, mixed with junior investigators who will undoubtedly lead this field over the coming decade. Presentations include findings from the only two ongoing clinical treatment trials for HGPS presented by trial Principle Investigator Monica E. Kleinman, MD, and other trial team experts will present development of new disease outcome measures using echocardiography and magnetic resonance imaging that may qualify as a viable HGPS biomarker. Future interventions also take the stage, with new data from prestigious investigators such as NIH Director Francis S. Collins, MD, PhD and NCI Distinguished Investigator Tom Misteli, PhD supporting treatment with RNA therapeutics, post-translational pathway-based intervention, and new data on CRISPR/Cas applied to HGPS mouse models. A session dedicated to the lessons learned from aging research and its overlaps with HGPS is moderated Judith Campisi, PhD, a world-renowned aging investigator. Based on prior meeting trends, we estimate over 200 attendees from 15 countries, 24 speakers and over 60 posters. Conclusion: Science presented at this meeting will greatly contribute to the next wave of discovery in Progeria and its relationships to aging and CVD in the general population.
The Progeria Research Foundation will conduct its ninth international scientific workshop on Progeria, a rare premature aging disease in which children develop accelerated atherosclerosis and die of heart attacks and strokes at an average age of 14.6 years (Cambridge, MA, September, 2018). The workshop will serve to facilitate collaborations and guide future basic, translational and clinical research on HGPS and its relationships to heart disease and aging. Because the disease- causing protein progerin is expressed in cells and tissues of normally aging individuals, the scientific advances and future strategies discussed at this workshop may have high relevance to normal aging and its associated cardiovascular disease, the leading cause of death in developed countries.
