Lentiviral vectors for gene therapy of Gaucher disease
Gaucher disease (GD), a recessive disorder characterised by hepatosplenomegaly, pancytopenia and skeletal complications, is caused by deficiency of the enzyme glucocerebrosidase (GC). GD leads to the accumulation of glucocerebrosides within macrophages, particularly in the liver and spleen. Current...
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University College London (University of London)
2015
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| Online Access: | http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.647316 |