AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

AAV8 Gene Therapy for Crigler-Najjar Syndrome in Macaques Elicited Transgene T Cell Responses That Are Resident to the Liver

Systemic delivery of adeno-associated viral (AAV) vectors has been evaluated for the treatment of several liver diseases, including homozygous familial hypercholesterolemia, ornithine transcarbamylase deficiency, and hemophilia. Here, we evaluated this approach for the treatment of Crigler-Najjar sy...

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Bibliographic Details
Main Authors: Jenny A. Greig, Roberto Calcedo, Leticia Kuri-Cervantes, Jayme M.L. Nordin, Jessica Albrecht, Erin Bote, Tamara Goode, Edward A. Chroscinski, Peter Bell, Laura K. Richman, Michael R. Betts, James M. Wilson
Format: Article
Language:English
Published: Elsevier 2018-12-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050118301086

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http://www.sciencedirect.com/science/article/pii/S2329050118301086

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