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Expression of a Secretable, Cell-Penetrating CDKL5 Protein Enhances the Efficacy of Gene Therapy for CDKL5 Deficiency Disorder.
Medici G, Tassinari M, Galvani G, Bastianini S, Gennaccaro L, Loi M, Mottolese N, Alvente S, Berteotti C, Sagona G, Lupori L, Candini G, Baggett HR, Zoccoli G, Giustetto M, Muotri A, Pizzorusso T, Nakai H, Trazzi S, Ciani E. Medici G, et al. Neurotherapeutics. 2022 Oct;19(6):1886-1904. doi: 10.1007/s13311-022-01295-8. Epub 2022 Sep 15. Neurotherapeutics. 2022. PMID: 36109452 Free PMC article.
Site-directed RNA repair of endogenous Mecp2 RNA in neurons.
Sinnamon JR, Kim SY, Corson GM, Song Z, Nakai H, Adelman JP, Mandel G. Sinnamon JR, et al. Proc Natl Acad Sci U S A. 2017 Oct 31;114(44):E9395-E9402. doi: 10.1073/pnas.1715320114. Epub 2017 Oct 16. Proc Natl Acad Sci U S A. 2017. PMID: 29078406 Free PMC article.
Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.
Cabanes-Creus M, Ginn SL, Amaya AK, Liao SHY, Westhaus A, Hallwirth CV, Wilmott P, Ward J, Dilworth KL, Santilli G, Rybicki A, Nakai H, Thrasher AJ, Filip AC, Alexander IE, Lisowski L. Cabanes-Creus M, et al. Mol Ther Methods Clin Dev. 2018 Nov 1;12:71-84. doi: 10.1016/j.omtm.2018.10.016. eCollection 2019 Mar 15. Mol Ther Methods Clin Dev. 2018. PMID: 30534580 Free PMC article.