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Targeted introduction and effective expression of hFIX at the AAVS1 locus in mesenchymal stem cells

Hemophilia B occurs due to a deficiency in human blood coagulation factor IX (hFIX). Currently, no effective treatment for hemophilia B has been identified, and gene therapy has been considered the most appropriate treatment. Mesenchymal stem cells (MSCs) have homing abilities and low immunogenicity...

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Detalles Bibliográficos
Autores principales: Li, Shu-Jun, Luo, Ying, Zhang, Le-Meng, Yang, Wei, Zhang, Guo-Gang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: D.A. Spandidos 2017
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5367337/
https://www.ncbi.nlm.nih.gov/pubmed/28112377
http://dx.doi.org/10.3892/mmr.2017.6131

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