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O'Geen H, Beitnere U, Garcia MS, Adhikari A, Cameron DL, Fenton TA, Copping NA, Deng P, Lock S, Halmai JANM, Villegas IJ, Liu J, Wang D, Fink KD, Silverman JL, Segal DJ. Transcriptional reprogramming restores UBE3A brain-wide and rescues behavioral phenotypes in an Angelman syndrome mouse model. Mol Ther 2023:S1525-0016(23)00013-8. [PMID: 36641623 DOI: 10.1016/j.ymthe.2023.01.013] [Reference Citation Analysis]
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Jagasia R, Bon C, Rasmussen SV, Badillo S, Tehler D, Buchy D, Berrera M, Prasad M, Terrigno M, Pandya NJ, Costa V, Wang C, Pedersen L, Miller MT, Dumong Erichsen K, Joenson L, Hipp J, Bonni A, Müller L, Brändli-baiocco A, Kremer T, Koller E, Hoener MC. Angelman syndrome patient neuron screen identifies a potent and selective clinical ASO targeting UBE3A-ATS with long lasting effect in cynomolgus monkey.. [DOI: 10.1101/2022.06.09.495066] [Cited by in Crossref: 1] [Cited by in F6Publishing: 1] [Article Influence: 1.0] [Reference Citation Analysis]
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