Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates

The potential host immune response to a nonself protein poses a fundamental challenge for gene therapies targeting recessive diseases. We demonstrate in both dogs and nonhuman primates that liver-directed gene transfer using an adeno-associated virus (AAV) vector in neonates induces a persistent sta...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Hinderer, Christian, Bell, Peter, Louboutin, Jean-Pierre, Zhu, Yanqing, Yu, Hongwei, Lin, Gloria, Choa, Ruth, Gurda, Brittney L, Bagel, Jessica, O'Donnell, Patricia, Sikora, Tracey, Ruane, Therese, Wang, Ping, Tarantal, Alice F, Casal, Margret L, Haskins, Mark E, Wilson, James M
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4817868/
https://ncbi.nlm.nih.gov/pubmed/26022732
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2015.99
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