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Augmentation of Transgene-Encoded Protein After Neonatal Injection of Adeno-Associated Virus Improves Hepatic Copy Number Without Immune Responses
BACKGROUND: Achieving persistent expression is a prerequisite for genetic therapies for inherited metabolic enzymopathies. Such disorders potentially could be treated with gene therapy shortly after birth to prevent pathology. However, rapid cell turnover leads to hepatic episomal vector loss, which...
| Autores principales: | , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
2015
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4540625/ https://www.ncbi.nlm.nih.gov/pubmed/26042522 http://dx.doi.org/10.1038/pr.2015.109 |