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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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: 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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
Assuntos:
Original Article
Acesso em linha: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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