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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Detalles Bibliográficos
Autores principales: Greig, Jenny A., Calcedo, Roberto, Kuri-Cervantes, Leticia, Nordin, Jayme M.L., Albrecht, Jessica, Bote, Erin, Goode, Tamara, Chroscinski, Edward A., Bell, Peter, Richman, Laura K., Betts, Michael R., Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6282099/
https://www.ncbi.nlm.nih.gov/pubmed/30547050
http://dx.doi.org/10.1016/j.omtm.2018.10.012

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6282099/
https://www.ncbi.nlm.nih.gov/pubmed/30547050
http://dx.doi.org/10.1016/j.omtm.2018.10.012

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