Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice

Sung Chul Jung, Ina P. Han, Advait Limaye, Ruian Xu, Monique P. Gelderman, Patricia Zerfas, Kamala Tirumalai, Gary J. Murray, Matthew J. During, Roscoe O. Brady, Pankaj Qasba

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Medicine & Life Sciences