Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector.
De Sabbata G, Boisgerault F, Guarnaccia C, Iaconcig A, Bortolussi G, Collaud F, Ronzitti G, Sola MS, Vidal P, Rouillon J, Charles S, Nicastro E, D'Antiga L, Ilyinskii P, Mingozzi F, Kishimoto TK, Muro AF.
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Mol Ther Methods Clin Dev. 2020 Nov 17;20:169-180. doi: 10.1016/j.omtm.2020.11.005. eCollection 2021 Mar 12.
Mol Ther Methods Clin Dev. 2020.
PMID: 33473356
Free PMC article.