Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

Vector capsid dose-dependent inflammation of transduced liver has limited the ability of adeno-associated virus (AAV) factor IX (FIX) gene therapy vectors to reliably convert severe to mild hemophilia B in human clinical trials. These trials also identified the need to understand AAV neutralizing an...

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Detalles Bibliográficos
Autores principales: Monahan, Paul E., Sun, Junjiang, Gui, Tong, Hu, Genlin, Hannah, William B., Wichlan, David G., Wu, Zhijian, Grieger, Joshua C., Li, Chengwen, Suwanmanee, Thipparat, Stafford, Darrel W., Booth, Carmen J., Samulski, Jade J., Kafri, Tal, McPhee, Scott W.J., Samulski, R. Jude
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc. 2015
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4326268/
https://www.ncbi.nlm.nih.gov/pubmed/25419787
http://dx.doi.org/10.1089/hum.2014.106

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