Platt, Frances M.

Lysosomal storage diseases [electronic resource] / Fran Platt. - London : Henry Stewart Talks, 2019. - 1 online resource (1 streaming video file (49 min.) : color, sound). - Autophagy and lysosomal storage diseases, 2056-452X . - Henry Stewart talks. Biomedical & life sciences collection. Autophagy and lysosomal storage diseases. .

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.

4891 Henry Stewart Talks


Lysosomal storage disorders.
Lysosomes.
Lysosomal Storage Diseases--drug therapy.
Lysosomal Storage Diseases--pathology.
Lysosomal Storage Diseases--therapy.
Lysosomes--metabolism.
Neurodegenerative Diseases.
Sphingolipids--metabolism.
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