Article

A Cross-Correction Gene Therapy Approach for CDKL5 Deficiency Disorder Improves the Pathological Phenotype of CDD Patient-Derived Cortical Organoids

2025-07-18

Abstract excerpt

Efficient delivery of biological material to the central nervous system (CNS) 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 of the gene therapy approach in a mouse model of CDKL5 Deficiency Disorder (CDD). Here, to compare the...

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Literature Corpus work: b5950c01-eb66-5a29-8a22-160a77863ada
DOI: 10.20944/preprints202507.1555.v1

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