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Long-term reproducible expression in human fetal liver hematopoietic stem cells with a UCOE-based lentiviral vector.

Hematopoietic Stem Cell (HSC) targeted gene transfer is an attractive treatment option for a number of hematopoietic disorders caused by single gene defects. However, extensive methylation of promoter sequences results in silencing of therapeutic gene expression. The choice of an appropriate promote...

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Autors principals: Niraja Dighe, Maroun Khoury, Citra Mattar, Mark Chong, Mahesh Choolani, Jianzhu Chen, Michael N Antoniou, Jerry K Y Chan
Format: Artigo
Idioma:Inglês
Publicat: Public Library of Science (PLoS) 2014-01-01
Col·lecció:PLoS ONE
Accés en línia:http://europepmc.org/articles/PMC4130605?pdf=render
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