EXPRESSION OF A SECRETABLE, CELL-PENETRATING CDKL5 PROTEIN ENHANCES THE EFFICACY OF AAV VECTOR-MEDIATED 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, Helen Rappe Baggett, Giovanna Zoccoli, Maurizio Giustetto, Alysson Renato Muotri, , Elisabetta Ciani
This study shows that using a secretable TATk-CDKL5 protein in AAV gene therapy improves CDKL5 replacement and functional outcomes in Cdkl5 KO mice compared to native CDKL5 expression.
bioRxiv · Jul 26, 2021 · PreprintNeuroscience