Proof-of-concept for a gene replacement approach to CDKL5 deficiency disorder
- PMID: 32203572
- PMCID: PMC7089659
- DOI: 10.1093/brain/awaa055
Proof-of-concept for a gene replacement approach to CDKL5 deficiency disorder
Abstract
This scientific commentary refers to ‘Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder’, by Gao etal. (doi:
Comment on
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Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder.Brain. 2020 Mar 1;143(3):811-832. doi: 10.1093/brain/awaa028. Brain. 2020. PMID: 32125365
References
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- Devinsky O, Verducci C, Thiele EA, Laux LC, Patel AD, Filloux F, et al.Open-label use of highly purified CBD (Epidiolex(R)) in patients with CDKL5 deficiency disorder and Aicardi, Dup15q, and Doose syndromes. Epilepsy Behav 2018; 86: 131–7. - PubMed
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- Gao Y, Irvine E, Eleftheriadou I, Jimenez Naranjo C, Bosch L, Glegola J, et al.Gene replacement ameliorates deficits in mouse and human models of cyclin-dependent kinase-like 5 disorder. Brain 2020; 143: 811–832. - PubMed
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