NeurosciencebioRxivPreprint
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…·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.
DOI 10.1101/2021.07.26.453746