Article

Gene therapy with AAV-CDKL5 vectors in models of CDKL5 disorder

2019-01-01

Abstract excerpt

CDKL5 disorder is a severe neurodevelopmental disorder caused by mutations in the X-linked cyclin-dependent kinase-like 5 (CDKL5) gene. It predominantly affects females that typically present with severe epileptic encephalopathy, intellectual disability, autistic features and motor dysfunction. Currently, there is no therapy apart from anti-epileptic drugs providing poor seizure management. To develop a gene repla...

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Literature Corpus work: 5c3c36a2-c5f4-548f-95cc-ade0ca55cec3
DOI: 10.25560/90058

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