Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism
基因疗法可恢复多巴胺转运蛋白的表达并改善婴儿帕金森病 iPSC 和小鼠模型中的病理
期刊:Science Translational Medicine
影响因子:15.8
doi:10.1126/scitranslmed.aaw1564
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, Julien Baruteau, Natalie Suff, Juan Antinao Diaz,