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...

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Detalles Bibliográficos
Autores principales: Tai, Denise S., Hu, Chuhong, Kim, Elizabeth H., Lipshutz, Gerald S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2015
Materias:
Article
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

Internet

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

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