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Keywords: neuronal ceroid lipofuscinosis
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Biochem Soc Trans (2010) 38 (6): 1484–1488.
Published: 24 November 2010
... therapy enzyme-replacement therapy gene therapy neuronal ceroid lipofuscinosis stem cell therapy Over the last two decades, our understanding of the soluble lysosomal forms of NCL has moved from pre-clinical studies to two complete Phase I clinical trials, with Phase II efficacy trials likely...