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.
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Neurotherapeutics. 2024 Sep;21(5):e00376. doi: 10.1016/j.neurot.2024.e00376. Epub 2024 Jun 14.
Neurotherapeutics. 2024.
PMID: 38876822
Free PMC article.