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...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Veröffentlicht in:Mol Ther
Hauptverfasser: 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
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2015
Schlagworte:
Original Article
Online Zugang: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
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!

Ähnliche Einträge