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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Publicat a:Mol Cell Pediatr
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: Springer Berlin Heidelberg 2018
Matèries:
Short Communication
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6236008/
https://ncbi.nlm.nih.gov/pubmed/30430274
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s40348-018-0086-1
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