Article

Development and characterisation of a zinc finger nuclease specific for the human beta-globin gene

2013-01-01

Abstract excerpt

β-thalassemia and sickle cell disease, which are caused by mutations in the β-globin gene, are two of the most common single gene disorders worldwide and the only available cure is allogeneic bone marrow transplantation that is limited by donor availability. Gene therapy, by delivery of a β-globin expression vector into autologous haematopoietic stem cells, is a valuable alternative but the technique is affected b...

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Literature Corpus work: 164ea542-f2d8-5691-9753-a5908799fbc1
DOI: 10.25560/17876

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