Gene Delivery of Activated Factor VII Using Alternative Adeno-Associated Virus Serotype Improves Hemostasis in Hemophiliac Mice with FVIII Inhibitors and Adeno-Associated Virus Neutralizing Antibodies

While therapeutic expression of coagulation factors from adeno-associated virus (AAV) vectors has been successfully achieved in patients with hemophilia, neutralizing antibodies to the vector and inhibitory antibodies to the transgene severely limit efficacy. Indeed, approximately 40% of mice transd...

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Pubblicato in:Hum Gene Ther
Autori principali: Sun, Junjiang, Hua, Baolai, Chen, Xiaojing, Samulski, Richard J., Li, Chengwen
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc. 2017
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Research Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5568595/
https://ncbi.nlm.nih.gov/pubmed/28478688
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2017.016
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