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Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1.
Torella L, Klermund J, Bilbao-Arribas M, Tamayo I, Andrieux G, Chmielewski KO, Vales A, Olagüe C, Moreno-Luqui D, Raimondi I, Abad A, Torrens-Baile J, Salido E, Huarte M, Hernaez M, Boerries M, Cathomen T, Zabaleta N, Gonzalez-Aseguinolaza G. Torella L, et al. Among authors: gonzalez aseguinolaza g. EMBO Mol Med. 2024 Jan;16(1):112-131. doi: 10.1038/s44321-023-00008-8. Epub 2024 Jan 5. EMBO Mol Med. 2024. PMID: 38182795 Free PMC article.
Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria.
Pañeda A, Lopez-Franco E, Kaeppel C, Unzu C, Gil-Royo AG, D'Avola D, Beattie SG, Olagüe C, Ferrero R, Sampedro A, Mauleon I, Hermening S, Salmon F, Benito A, Gavira JJ, Cornet ME, del Mar Municio M, von Kalle C, Petry H, Prieto J, Schmidt M, Fontanellas A, González-Aseguinolaza G. Pañeda A, et al. Hum Gene Ther. 2013 Dec;24(12):1007-17. doi: 10.1089/hum.2013.166. Hum Gene Ther. 2013. PMID: 24070415
150 results