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Modeling Antisense Oligonucleotide Therapy in MECP2 Duplication Syndrome Human Ipsc-Derived Neurons Reveals Gene Expression Programs Responsive to MeCP2 Levels

Sameer S. Bajikar,Yehezkel Sztainberg,Alexander J. Trostle, Harini P. Tirumala,Ying-Wooi Wan, Caroline L. Harrop,Jesse D. Bengtsson,Claudia M. B. Carvalho,Davut Pehlivan,Bernhard Suter,Jeffrey L. Neul,Zhandong Liu,Paymaan Jafar-Nejad,Frank Rigo,Huda Y. Zoghbi

HUMAN MOLECULAR GENETICS（2024）

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关键词
genetic syndromes,copy-number gain,iPSC derived neurons,transcriptomics,genomic disorder
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