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Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder

Gregory Voronin, Jana Narasimhan, Jamila Gittens, Josephine Sheedy, Philip Lipari, Melinda Peters, Steven J. DeMarco, Liangxian Cao, Yakov Varganov, Min Jung Kim, Lisset Pear, Eman Fotouh, Supriya Sinha, Balmiki Ray, Michael C. Wu, Padmaja Yalamanchili, Christopher Southgate, Joseph Pick, Khalil Saadipour, Stephen P. Jung, J.M. Lee, Anna Mollin, Ellen Welch, Zhijian Wu, Marla Weetall

2024Molecular Therapy11 citationsDOIOpen Access PDF

Topics & Concepts

Genetic enhancementMedicineGeneBiologyGeneticsBioinformaticsGenetics and Neurodevelopmental DisordersAutism Spectrum Disorder ResearchGenomic variations and chromosomal abnormalities
Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder | Litcius