The Progeria Research Foundation 8th International Scientific Workshop "Across the Table, Around the Globe"

项目总结

Project Summary 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 2016 PRF International Scientific Workshop on Progeria will be held in Cambridge, MA on May 2-4, 2016. This will be the 8th 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, 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 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 comprehensive “baseline” session summarizing key clinical and basic science discoveries in HGPS and their relationships to aging and CVD. This will lay the foundation for more in-depth discussion 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. Day two will include findings from the only two ongoing clinical treatment trials for HGPS, and ongoing proteomic studies to identify biomarkers of disease and to quantify farnesylated progerin, the HGPS-causing mutant protein. Future interventions then take center stage, with new data supporting 7 pharmacologic interventions, 3 RNA therapeutics, and telomere- based treatment strategies. A cutting edge basic science session then highlights the role of lamin A in disease and aging, including induced pluripotent stem cell research. The wrap-up will be led by Francis Collins, MD, PhD (NIH), who discovered the HGPS mutation, Judith Campisi, PhD (Buck Inst.), a world-renowned aging investigator, and Mark Kieran, MD, PhD (Harvard U.), Principal Investigator for HGPS clinical trials. They will facilitate an interactive discussion of what the basic and clinical scientists have gained from the meeting, and what the attendees will bring forward into their future research. We estimate over 200 attendees from 15 countries, 26 speakers and 60 posters. Conclusion: Science presented at this meeting represents the next wave of discovery in Progeria and its relationships to aging and CVD in the geriatric population.