Enhanced Factor IX Activity following Administration of AAV5-R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs

Gene therapy for severe hemophilia B is advancing and offers sustained disease amelioration with a single treatment. We have reported the efficacy and safety of AMT-060, an investigational gene therapy comprising an adeno-associated virus serotype 5 capsid encapsidating the codon-optimized wild-type...

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Publicado en:Mol Ther Methods Clin Dev
Autores principales: Spronck, Elisabeth A., Liu, Ying Poi, Lubelski, Jacek, Ehlert, Erich, Gielen, Sander, Montenegro-Miranda, Paula, de Haan, Martin, Nijmeijer, Bart, Ferreira, Valerie, Petry, Harald, van Deventer, Sander J.
Formato: Artigo
Lenguaje:Inglês
Publicado: American Society of Gene & Cell Therapy 2019
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC6834974/
https://ncbi.nlm.nih.gov/pubmed/31709273
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2019.09.005
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