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Gene therapy for inborn errors of liver metabolism: progress towards clinical applications
The treatment for inborn errors of liver metabolism is based on dietary, drug, and cell therapies (orthotopic liver transplantation). However, significant morbidity and mortality still remain, and alternative strategies are needed. Gene replacement therapy has the potential of providing a definitive...
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| Formato: | Texto |
| Lenguaje: | English |
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BioMed Central
2008
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2603013/ https://www.ncbi.nlm.nih.gov/pubmed/19490653 http://dx.doi.org/10.1186/1824-7288-34-2 |