Exploring the potential for gene therapy in multiple forms of neuronal ceroid lipofuscinosis (Batten disease)
The neuronal ceroid lipofuscinoses (NCLs) are a group of inherited lysosomal storage disorders that cause profound neurodegeneration and predominantly affect children. Currently, there are no effective treatments available for any form of NCL. This thesis focuses on novel applications of gene therap...
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King's College London (University of London)
2016
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| Online Access: | http://ethos.bl.uk/OrderDetails.do?uin=uk.bl.ethos.700771 |