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Author(s) -
Tommaso Vannocci,
Hitoshi Kurata,
Josu de la Fuente,
Irene A Roberts,
Andrew C G Porter
Publication year - 2014
Publication title -
the journal of gene medicine
Language(s) - Uncategorized
Resource type - Journals
SCImago Journal Rank - 0.689
H-Index - 91
eISSN - 1521-2254
pISSN - 1099-498X
DOI - 10.1002/jgm.2758
Subject(s) - citation , library science , computer science , information retrieval , world wide web
Mutations in the β-globin gene (HBB) cause haemoglobinopathies where current treatments have serious limitations. Gene correction by homologous recombination (HR) is an attractive approach to gene therapy for such diseases and is stimulated by gene-specific endonucleases, including zinc finger nucleases (ZFNs). Customised nucleases targeting HBB have previously been shown to promote HR-mediated HBB modification in 0.3–60% of drug-selected cells, although frequencies among unselected cells, more relevant to the goal of correcting HBB in primary stem cells, have not been reported.