Intragenic integration in DLC1 sustains factor VIII expression in primary human cells without insertional oncogenicity

Techniques enabling precise genome modifications enhance the safety of gene-based therapy. DLC1 is a hot spot for phiC31 integrase-mediated transgene integration in vitro and in vivo. Here we show that integration of a coagulation factor VIII transgene into intron 7 of DLC1 supports durable expressi...

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Detalles Bibliográficos
Autores principales: Sivalingam, J, Phan, T T, Kon, O L
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3975812/
https://www.ncbi.nlm.nih.gov/pubmed/24553346
http://dx.doi.org/10.1038/gt.2014.11

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