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| Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow D Pan, R Gunther, W Duan, S Wendell, W Kaemmerer, T Kafri, IM Verma, ... Molecular Therapy 6 (1), 19-29, 2002 | 160 | 2002 |
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| Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial J Diaz-Manera, PS Kishnani, H Kushlaf, S Ladha, T Mozaffar, V Straub, ... The Lancet Neurology 20 (12), 1012-1026, 2021 | 138 | 2021 |
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