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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,

Molecular Therapy · 2024 · 11 citations · 22 references

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Local generation of glia is a major astrocyte source in postnatal cortex

Woo‐Ping Ge, Atsushi Miyawaki, Fred H. Gage et al. · Nature · 2012 · 512 citations · Full text

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Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area

Sebastian Kügler, Mathias Bähr · Gene Therapy · 2003 · 422 citations

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The CDKL5 disorder is an independent clinical entity associated with early-onset encephalopathy

Stephanie Fehr, Meredith Wilson, Jenny Downs et al. · European Journal of Human Genetics · 2012 · 314 citations

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Loss of CDKL5 disrupts kinome profile and event-related potentials leading to autistic-like phenotypes in mice

I-Ting Judy Wang, Megan Allen, Darren Goffin et al. · Proceedings of the National Academy of Sciences · 2012 · 233 citations · Full text

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Mapping Pathological Phenotypes in a Mouse Model of CDKL5 Disorder

Elena Amendola, Yang Zhan, Camilla Mattucci et al. · PLoS ONE · 2014 · 217 citations · Full text

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