CRISPR/Cas9 Beta-globin Gene Targeting in Human Hematopoietic Stem Cells

The β-hemoglobinopathies, including sickle cell disease (SCD) and β-thalassemia, are caused by mutations in the β-globin gene (HBB) and affect millions of people worldwide. A curative strategy for the β-hemoglobinopathies would be ex vivo gene correction in patient-derived hematopoietic stem cells (...

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Bibliografische gegevens
Gepubliceerd in:Nature
Hoofdauteurs: Dever, Daniel P., Bak, Rasmus O., Reinisch, Andreas, Camarena, Joab, Washington, Gabriel, Nicolas, Carmencita E., Pavel-Dinu, Mara, Saxena, Nivi, Wilkens, Alec B., Mantri, Sruthi, Uchida, Nobuko, Hendel, Ayal, Narla, Anupama, Majeti, Ravindra, Weinberg, Kenneth I., Porteus, Matthew H.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2016
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5898607/
https://ncbi.nlm.nih.gov/pubmed/27820943
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nature20134
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