? The Alpha-1 Foundation (A1F) and the American Association for the Study of Liver Diseases (AASLD) are organizing a Basic Research Single Topic Conference entitled """"""""Alpha-1 Antitrypsin Deficiency and Other Liver Diseases Caused by Aggregated Proteins"""""""" on January 26-28, 2006. This conference focuses on an important, emerging topic: the molecular pathogenesis and therapy for Alpha-1 Antitrypsin (AAT) Deficiency and other inherited metabolic disorder of protein folding. The conference will include presentations by internationally recognized experts on the molecular pathogenesis of AAT Deficiency and other liver diseases associated with aggregated proteins. A primary goal is to stimulate a deeper understanding of the molecular mechanisms underlying these liver diseases to review and explore potential therapeutic approaches. This Basic Research Single Topic Conference is the culmination of an ongoing collaboration between the A1F and AASLD designed to to increase awareness of AAT Deficiency as well as increase the overall understanding of the underlying pathogenesis for a number of different liver diseases. This conference builds upon several previous symposia and conferences that the A1F and AASLD have held since 2000, including Alpha-1 Antitrypsin Deficiency and Other Conformational Diseases (June, 2000) and symposia during the 2003 and 2004 AASLD Annual Meetings on the Hepatotoxicity associated with Alpha-1 and Other Liver Diseases, and Insights into Metabolic Diseases. The 2006 conference will examine the recent contributions from basic research over the past 5 years that have improved our understanding of the mechanisms by which glycoproteins such as alpha-1 antitrypsin are correctly folded within the endoplasmic reticulum. The conference is also predicated by clinical data that is beginning to emerge that may explain the molecular basis for why some individuals with AAT Deficiency get liver disease and some do not. Another focus of the conference is the significant work in translational liver research that combines gene transfer technology and cell biology to create and evaluate hepatocyte transplantation therapeutic strategies for end stage liver diseases. The identified goals of the conference are to: 1) Understand the molecular biology of protein folding, the role of the protein degradation system, and the consequences of misfolding of specific proteins seen in several clinical disorders; 2) Discuss the current concepts of the molecular mechanisms responsible for liver injury in patients with Alpha-1 Antitrypsin Deficiency; 3) Explain the current molecular approaches underway to develop effective therapies to treat the liver manifestations of Alpha-1 Antitrypsin Deficiency and other disorders involving protein misfolding. ? ?
| Perlmutter, David H; Brodsky, Jeffrey L; Balistreri, William F et al. (2007) Molecular pathogenesis of alpha-1-antitrypsin deficiency-associated liver disease: a meeting review. Hepatology 45:1313-23 |
