Development and characterisation of a zinc finger nuclease specific for the human beta-globin gene
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 e...
| Main Author: | |
|---|---|
| Other Authors: | |
| Published: |
Imperial College London
2013
|
| Subjects: | |
| Online Access: | http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.631177 |