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Ann Transl Med. 2015 May;3(Suppl 1):S28. doi: 10.3978/j.issn.2305-5839.2015.02.28.

Gene therapy for GM1 gangliosidosis: challenges of translational medicine.

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1 NIHR Biomedical Research Unit, Royal Brompton Hospital, London & Imperial College, London, UK ; 2 Department of Neurology, John Radcliffe Hospital, Oxford, UK.

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