Preclinical studies of gene replacement therapy for CDKL5 deficiency disorder

作者：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 · 发表于：Molecular Therapy · 年份：2024 · DOI：10.1016/j.ymthe.2024.07.012 · 被引用次数：12 · 研究领域：Genetics and Neurodevelopmental Disorders、Autism Spectrum Disorder Research、Genomic variations and chromosomal abnormalities