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Less Is More: Substrate Reduction Therapy for Lysosomal Storage Disorders
Lysosomal storage diseases (LSDs) are a group of rare, life-threatening genetic disorders, usually caused by a dysfunction in one of the many enzymes responsible for intralysosomal digestion. Even though no cure is available for any LSD, a few treatment strategies do exist. Traditionally, efforts ha...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2016
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4964441/ https://www.ncbi.nlm.nih.gov/pubmed/27384562 http://dx.doi.org/10.3390/ijms17071065 |