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Gene correction of HBB mutations in CD34(+) hematopoietic stem cells using Cas9 mRNA and ssODN donors

BACKGROUND: β-Thalassemia is an inherited hematological disorder caused by mutations in the human hemoglobin beta (HBB) gene that reduce or abrogate β-globin expression. Although lentiviral-mediated expression of β-globin and autologous transplantation is a promising therapeutic approach, the risk o...

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Detalles Bibliográficos
Autores principales:	Antony, Justin S., Latifi, Ngadhnjim, Haque, A. K. M. Ashiqul, Lamsfus-Calle, Andrés, Daniel-Moreno, Alberto, Graeter, Sebastian, Baskaran, Praveen, Weinmann, Petra, Mezger, Markus, Handgretinger, Rupert, Kormann, Michael S. D.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Springer Berlin Heidelberg 2018
Materias:	Short Communication
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6236008/ https://www.ncbi.nlm.nih.gov/pubmed/30430274 http://dx.doi.org/10.1186/s40348-018-0086-1

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