Characterization of Gene Alterations following Editing of the β-Globin Gene Locus in Hematopoietic Stem/Progenitor Cells

The use of engineered nucleases combined with a homologous DNA donor template can result in targeted gene correction of the sickle cell disease mutation in hematopoietic stem and progenitor cells. However, because of the high homology between the adjacent human β- and δ-globin genes, off-target clea...

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Veröffentlicht in:Mol Ther
Hauptverfasser: Long, Joseph, Hoban, Megan D., Cooper, Aaron R., Kaufman, Michael L., Kuo, Caroline Y., Campo-Fernandez, Beatriz, Lumaquin, Dianne, Hollis, Roger P., Wang, Xiaoyan, Kohn, Donald B., Romero, Zulema
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society of Gene & Cell Therapy 2018
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Original Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5835144/
https://ncbi.nlm.nih.gov/pubmed/29221806
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.11.001
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