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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Detaylı Bibliyografya
Yayımlandı:Hum Gene Ther
Asıl Yazarlar: 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
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Mary Ann Liebert, Inc. 2015
Konular:
Research Articles
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC4326268/
https://ncbi.nlm.nih.gov/pubmed/25419787
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2014.106
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