Expression of a Secretable, Cell-Penetrating CDKL5 Protein Enhances the Efficacy of Gene Therapy for CDKL5 Deficiency Disorder
Giorgio Medici,Marianna Tassinari,Giuseppe Galvani,Stefano Bastianini,Laura Gennaccaro,Manuela Loi,Nicola Mottolese,Sara Alvente,Chiara Berteotti,Giulia Sagona,Leonardo Lupori,Giulia Candini,Helen Rappe Baggett,Giovanna Zoccoli,Maurizio Giustetto,Alysson Muotri,Tommaso Pizzorusso,Hiroyuki Nakai,Stefania Trazzi,Elisabetta Ciani Neurotherapeutics（2022）
关键词
AAV gene therapy,Cross-correction,CDKL5,Brain disorder,Mouse model
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