A cross-correction gene therapy approach for CDKL5 deficiency disorder improves the pathological phenotype of CDD patient-derived cortical organoids
Giorgio Medici, Angelica Marina Bove, Stefania Trazzi, Francesca Puppo et autres
Efficient delivery of biological material to the central nervous system remains a key limitation of conventional gene therapies. Recently, we developed a novel strategy based on a secretable and cell-penetrating TATk-CDKL5 fused protein which enhances the brain biodistribution and the therapeutic efficiency …
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Frontiers in Bioengineering and Biotechnology