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Neonatal Gene Therapy for Hemophilia B by a Novel Adenovirus Vector Showing Reduced Leaky Expression of Viral Genes

Gene therapy during neonatal and infant stages is a promising approach for hemophilia B, a congenital disorder caused by deficiency of blood coagulation factor IX (FIX). An adenovirus (Ad) vector has high potential for use in neonatal or infant gene therapy for hemophilia B due to its superior trans...

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Detalles Bibliográficos
Autores principales:	Iizuka, Shunsuke, Sakurai, Fuminori, Tachibana, Masashi, Ohashi, Kazuo, Mizuguchi, Hiroyuki
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2017
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5552065/ https://www.ncbi.nlm.nih.gov/pubmed/28828393 http://dx.doi.org/10.1016/j.omtm.2017.07.001

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