Article

Epigenetic Reactivation of CDKL5 Rescues CDKL5 Deficiency Disorder

2025-08-13

Abstract excerpt

<title>Abstract</title> <p>CDKL5 deficiency disorder (CDD) is a severe X-linked neurodevelopmental condition characterized by early-onset epilepsy, intellectual disability, and motor dysfunction. Here, we present a dual-effector CRISPR-based epigenome editing platform that enables targeted reactivation of the silenced <italic>CDKL5</italic> allele. Using a split dCas9 system compatible with AAV9 delivery, we achi...

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Literature Corpus work: 332c888d-2bbc-5617-a988-045f89d408b3
DOI: 10.21203/rs.3.rs-7095603/v1

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