This conference has been deferred to 2023 due to the ongoing impact of the COVID-19 pandemic. Please check back soon for the 2023 schedule.
With more than 80 rare inborn diseases of the lysosome and lysosome-related organelles, the lysosome is crucially implicated in several widespread diseases as well as numerous pathological processes - not least, neurodegeneration and defense. Advances in cell biology, informed in part through intensive research into lysosomal diseases, has shown the central role of the lysosome in metabolism: no longer a lowly body rich in acid hydrolases engaged solely in the constitutive recycling of spent components, this near-ubiquitous organelle has functions in survival, proliferation, energy-sensing and the adaptive provision of materials essential for manufacture. In essence, the lysosome is now recognized as a highly dynamic structure subject to regulatory control mechanisms that operate at the heart of the cellular economy.
With the striking success of cell-targeted enzyme therapy to augment the defective acid hydrolase in Gaucher disease, radical correction of other lysosomal diseases is a powerful ideal. Hitherto the orphan drug legislation has encouraged massive investment predicated on these ultra-rare diseases but the real-world outcomes of approved drugs are often less than promising: efficacy varies depending on the approach the intrinsic complexities of each disease. Exorbitant costs deny access to needy patients globally also call the extreme capitalist model into moral question. There is a pressing need for effective measures that build on scientific research that will solve genuine and persistent mechanistic hurdles.
The 2021 Gordon Research Conference on Lysosomal Diseases will cover the latest discoveries that advance knowledge about basic lysosomal function; how molecular pathways are affected in lysosomal diseases - and how these inform more universal application in biology and medicine. Whenever possible, advances in science will be linked to a better understanding of the clinical phenotype and authentic therapeutic discovery. We have speakers who will address additional roles of the lysosome in the complex control of innate as well as adaptive immunity and where lysosomal proteins are mutated, dysregulation of immune mechanisms with loss of tolerance. This conference is designed to attract the widest participation from molecular cell biologists, geneticists, clinical investigators, biochemists and those with an interest in immunity and inflammation. Attendance will reward those interested in the rare treasure revealed by the study of lysosomal diseases: beyond new insights into the role of this organelle in major human disorders, emphasis will be placed on the latest initiatives in translational science and scrutinizing innovative concepts for treatment.