Published in
American Association for the Advancement of Science, Science Translational Medicine, 594(13), 2021
DOI: 10.1126/scitranslmed.aaw1564
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Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism
Journal article published in 2021 by
Joanne Ng 
,
Serena Barral ,
Carmen De La Fuente Barrigon,
Gabriele Lignani,
Fatma A. Erdem ,
Rebecca Wallings ,
Riccardo Privolizzi ,
Giada Rossignoli,
Haya Alrashidi ,
Sonja Heasman ,
Esther Meyer,
Adeline Ngoh ,
Simon Pope ,
Rajvinder Karda ,
Dany Perocheau
and other authors.
,
Serena Barral ,
Carmen De La Fuente Barrigon,
Gabriele Lignani,
Fatma A. Erdem ,
Rebecca Wallings ,
Riccardo Privolizzi ,
Giada Rossignoli,
Haya Alrashidi ,
Sonja Heasman ,
Esther Meyer,
Adeline Ngoh ,
Simon Pope ,
Rajvinder Karda ,
Dany Perocheau
and other authors.
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Abstract
Viral vectors restore dopamine transporter function and ameliorate neuropathology in iPSC-derived neurons and a mouse model of infantile parkinsonism.