Lysosomal storage diseases [electronic resource] / Fran Platt.
Material type:
FilmSeries: Henry Stewart talksBiomedical & life sciences collection. Autophagy and lysosomal storage diseases: Publisher: London : Henry Stewart Talks, 2019Description: 1 online resource (1 streaming video file (49 min.) : color, sound)Subject(s): Lysosomal storage disorders | Lysosomes | Lysosomal Storage Diseases -- drug therapy | Lysosomal Storage Diseases -- pathology | Lysosomal Storage Diseases -- therapy | Lysosomes -- metabolism | Neurodegenerative Diseases | Sphingolipids -- metabolismOnline resources: Click here to access online | Series 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.
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Mode of access: World Wide Web.