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Mucopolysaccharidoses type I gene therapy
Mucopolysaccharidoses type I (MPS I) is an inherited metabolic disease characterized by a malfunction of the α‐l‐iduronidase (IDUA) enzyme leading to the storage of glycosaminoglycans in the lysosomes. This disease has longtime been studied as a therapeutic target for those studying gene therapy and...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley & Sons, Inc.
2021
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8525653/ https://www.ncbi.nlm.nih.gov/pubmed/34189746 http://dx.doi.org/10.1002/jimd.12414 |