A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency

Julie Tordo, Claire O'Leary, André S. L. M. Antunes, Nuria Palomar, Patrick Aldrin-Kirk, Mark Basche, Antonette Bennett, Zelpha D'Souza, Hélène Gleitz, Annie Godwin, Rebecca J. Holley, Helen Parker, Ai Yin Liao, Paul Rouse, Amir Saam Youshani, Larbi Dridi, Carla Martins, Thierry Levade, Kevin B. Stacey, Daniel M. DavisAdam Dyer, Nathalie Clément, Tomas Björklund, Robin R. Ali, Mavis Agbandje-McKenna, Ahad A. Rahim, Alexey Pshezhetsky, Simon N. Waddington, R. Michael Linden, Brian W. Bigger, Els Henckaerts

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Biochemistry, Genetics and Molecular Biology

Neuroscience