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  -