Targeted Gene Addition to a Safe Harbor locus in human CD34(+) Hematopoietic Stem Cells for Correction of X-linked Chronic Granulomatous Disease

Gene therapy with genetically modified human CD34+ hematopoietic stem cells (HSCs) may be safer using targeted integration (TI) of transgenes into a genomic ‘safe harbor’ site than random viral integration. We demonstrate that temporally optimized delivery of zinc finger nuclease mRNA via electropor...

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Detalles Bibliográficos
Autores principales: De Ravin, Suk See, Reik, Andreas, Liu, Pei-Qi, Li, Linhong, Wu, Xiaolin, Su, Ling, Raley, Castle, Theobald, Narda, Choi, Uimook, Song, Alexander H., Chan, Andy, Pearl, Jocelynn R., Paschon, David E., Lee, Janet, Newcombe, Hannah, Koontz, Sherry, Sweeney, Colin, Shivak, David A., Zarember, Kol A., Peshwa, Madhusudan V., Gregory, Philip D., Urnov, Fyodor D., Malech, Harry L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2016
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4824656/
https://www.ncbi.nlm.nih.gov/pubmed/26950749
http://dx.doi.org/10.1038/nbt.3513

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