CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
About this item
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Author / Creator
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. and Porteus, Matthew H.
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
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Publication information
Publisher
London: Nature Publishing Group UK
Subjects
More information
Scope and Contents
Contents
The β-haemoglobinopathies, such as sickle cell disease and β-thalassaemia, are caused by mutations in the β-globin (
HBB
) gene and affect millions of people worldwide.
Ex vivo
gene correction in patient-derived haematopoietic stem cells followed by autologous transplantation could be used to cure β-haemoglobinopathies. Here we present...
Alternative Titles
Full title
CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells
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Record Identifier
TN_cdi_proquest_miscellaneous_1837284274
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_1837284274
Other Identifiers
ISSN
0028-0836,1476-4687
E-ISSN
1476-4687
DOI
10.1038/nature20134
