Genetic Substrate Reduction Therapy: A Promising Approach for Lysosomal Storage Disorders
Lysosomal storage diseases are a group of rare genetic disorders characterized by the accumulation of storage molecules in late endosomes/lysosomes. Most of them result from mutations in genes encoding for the catabolic enzymes that ensure intralysosomal digestion. Conventional therapeutic options i...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2016-11-01
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| Series: | Diseases |
| Subjects: | |
| Online Access: | http://www.mdpi.com/2079-9721/4/4/33 |