Open Access UCL Research: Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy
discovery.ucl.ac.uk/id/eprint/10...
discovery.ucl.ac.uk
Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy
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UCL Discovery
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