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MeCP2 gene therapy ameliorates disease phenotype in mouse model for Pitt Hopkins syndrome.
Dennys CN, Vermudez SAD, Deacon RJM, Sierra-Delgado JA, Rich K, Zhang X, Buch A, Weiss K, Moxley Y, Rajpal H, Espinoza FD, Powers S, Ávila AS, Gogliotti RG, Cogram P, Niswender CM, Meyer KC. Dennys CN, et al. Neurotherapeutics. 2024 Sep;21(5):e00376. doi: 10.1016/j.neurot.2024.e00376. Epub 2024 Jun 14. Neurotherapeutics. 2024. PMID: 38876822 Free PMC article.