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Gene Therapy for Inherited Metabolic Diseases
Over the last two decades, gene therapy has been successfully translated to many rare diseases. The number of clinical trials is rapidly expanding and some gene therapy products have now received market authorisation in the western world. Inherited metabolic diseases (IMD) are orphan diseases freque...
| Autores principales: | , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Sciendo
2020
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8518100/ https://www.ncbi.nlm.nih.gov/pubmed/33554501 http://dx.doi.org/10.34763/jmotherandchild.20202402si.2004.000009 |