Lysosomal storage diseases
| Main Author: | |
|---|---|
| Format: | eBook |
| Language: | English |
| Published: |
London
Henry Stewart Talks
2019, 2019
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| Series: | Autophagy and lysosomal storage diseases
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| Subjects: | |
| Online Access: | |
| Collection: | Henry Stewart Talks - Collection details see MPG.ReNa |
Table of 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