Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism

Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism

Most inherited neurodegenerative disorders are incurable, and often only palliative treatment is available. Precision medicine has great potential to address this unmet clinical need. We explored this paradigm in dopamine transporter deficiency syndrome (DTDS), caused by biallelic loss-of-function m...

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Päätekijät: Ng, J, Barral, S, De La Fuente Barrigon, C, Lignani, G, Erdem, FA, Wallings, R, Privolizzi, R, Rossignoli, G, Alrashidi, H, Heasman, S, Meyer, E, Ngoh, A, Pope, S, Karda, R, Perocheau, D, Baruteau, J, Suff, N, Antinao Diaz, J, Schorge, S, Vowles, J, Marshall, LR, Cowley, SA, Sucic, S, Freissmuth, M, Counsell, JR, Wade-Martins, R, Heales, SJR, Rahim, AA, Bencze, M, Waddington, SN, Kurian, MA
Aineistotyyppi: Journal article
Kieli:English
Julkaistu: American Association for the Advancement of Science 2021

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