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

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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Bibliographic Details
Main Authors: Shunsuke Iizuka, Fuminori Sakurai, Masashi Tachibana, Kazuo Ohashi, Hiroyuki Mizuguchi
Format: Article
Language:English
Published: Elsevier 2017-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
adenovirus vector
hemophilia B
gene therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050117300840

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