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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Iizuka, Shunsuke, Sakurai, Fuminori, Tachibana, Masashi, Ohashi, Kazuo, Mizuguchi, Hiroyuki
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5552065/
https://ncbi.nlm.nih.gov/pubmed/28828393
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2017.07.001
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