Lentiviral gene therapy vector with UCOE stably restores function in iPSC-derived neutrophils of a CDG patient

A recent gamma-retroviral clinical Chronic Granulomatous Disease (CGD) gene therapy (GT) trial achieved proof-of-concept but was accompanied by activation of oncogenes and transgene silencing. The ubiquitous chromatin opening element (UCOE) comprises the sequences of two divergently oriented house-k...

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Publicado no:Matters (Zur)
Main Authors: Haenseler, Walther, Kuzmenko, Elena, Smalls-Mantey, Adjoa, Browne, Cathy, Seger, Reinhard, James, William S, Cowley, Sally A, Reichenbach, Janine, Siler, Ulrich
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6472893/
https://ncbi.nlm.nih.gov/pubmed/31008103
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.19185/matters.201805000005
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