TY - ADVS AU - Platt,Frances M. TI - Lysosomal storage diseases T2 - Autophagy and lysosomal storage diseases, PY - 2019/// CY - London PB - Henry Stewart Talks KW - Lysosomal storage disorders KW - Lysosomes KW - Lysosomal Storage Diseases KW - drug therapy KW - pathology KW - therapy KW - metabolism KW - Neurodegenerative Diseases KW - Sphingolipids N1 - Animated audio-visual presentation with synchronized narration; Title from title frames; Contents: Lysosomes are multifunctional -- Lysosome related organelles -- Complexity & functions of lysosomal proteins -- Lysosomal storage diseases -- Sphingolipid lysosomal storage diseases as an example -- Lysosomal diseases: multimorbidity disorders -- Pathogenic cascade beyond the Lysosomal System -- Specific therapies approved for some diseases -- Molecular mechanisms and therapeutic approaches of LSDs -- Lysosomal enzymes -- ERTs: approved or in advanced clinical trials -- Approved small molecules: SRT & EET -- Multiple gene therapy trials: enzyme deficiencies -- Cellular organelles targeted by LSD therapies -- Lysosomal & neurodegenerative diseases; Access restricted to subscribers UR - https://hstalks.com/bs/3912/ UR - https://hstalks.com/bs/p/1041/ ER -