Engineered tRNAs efficiently suppress CDKL5 premature termination codons

Engineered tRNAs efficiently suppress CDKL5 premature termination codons

Abstract The CDKL5 deficiency disorder (CDD) is a severe neurodevelopmental disorder characterized by early-onset epilepsy, intellectual disability, motor and visual dysfunctions. The causative gene is CDKL5, which codes for a kinase required for brain development. There is no cure for CDD patients;...

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Main Authors: Stefano Pezzini, Aurora Mustaccia, Pierre Aboa, Giorgia Faustini, Alessio Branchini, Mirko Pinotti, Angelisa Frasca, Joseph J. Porter, John D. Lueck, Nicoletta Landsberger
Format: Article
Language:English
Published: Nature Portfolio 2024-12-01
Series:Scientific Reports
Online Access:https://doi.org/10.1038/s41598-024-82766-0
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https://doi.org/10.1038/s41598-024-82766-0

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