Llwytho...
Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I
Lysosomal storage diseases (LSDs) are rare inherited metabolic disorders characterized by a dysfunction in lysosomes, leading to waste material accumulation and severe organ damage. Enzyme replacement therapy (ERT) and haematopoietic stem cell transplant (HSCT) have been exploited as potential treat...
Wedi'i Gadw mewn:
| Cyhoeddwyd yn: | J Inherit Metab Dis |
|---|---|
| Prif Awduron: | , , , , |
| Fformat: | Artigo |
| Iaith: | Inglês |
| Cyhoeddwyd: |
Springer Netherlands
2017
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| Pynciau: | |
| Mynediad Ar-lein: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5500670/ https://ncbi.nlm.nih.gov/pubmed/28560469 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s10545-017-0052-4 |
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