TY - ADVS TI - Lysosomal storage diseases AU - Platt, Fran PY - 2019 DA - 2019/03/31 UR - https://hstalks.com/bs/3912/ AB - 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 DB - The Biomedical & Life Sciences Collection SN - 2056-452X ID - 4891 PB - Henry Stewart Talks ER -