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Page 1
Ancestral library identifies conserved reprogrammable liver motif on AAV capsid.
Zinn E, Unzu C, Schmit PF, Turunen HT, Zabaleta N, Sanmiguel J, Fieldsend A, Bhatt U, Diop C, Merkel E, Gurrala R, Peacker B, Rios C, Messemer K, Santos J, Estelien R, Andres-Mateos E, Wagers AJ, Tipper C, Vandenberghe LH. Zinn E, et al. Among authors: unzu c. Cell Rep Med. 2022 Nov 15;3(11):100803. doi: 10.1016/j.xcrm.2022.100803. Epub 2022 Nov 2. Cell Rep Med. 2022. PMID: 36327973 Free PMC article.
Porphobilinogen deaminase over-expression in hepatocytes, but not in erythrocytes, prevents accumulation of toxic porphyrin precursors in a mouse model of acute intermittent porphyria.
Unzu C, Sampedro A, Mauleón I, Vanrell L, Dubrot J, de Salamanca RE, González-Aseguinolaza G, Melero I, Prieto J, Fontanellas A. Unzu C, et al. J Hepatol. 2010 Mar;52(3):417-24. doi: 10.1016/j.jhep.2009.09.003. Epub 2009 Sep 23. J Hepatol. 2010. PMID: 19815305 Free article.
Gene therapy for liver diseases - progress and challenges.
Zabaleta N, Unzu C, Weber ND, Gonzalez-Aseguinolaza G. Zabaleta N, et al. Among authors: unzu c. Nat Rev Gastroenterol Hepatol. 2023 May;20(5):288-305. doi: 10.1038/s41575-022-00729-0. Epub 2023 Jan 16. Nat Rev Gastroenterol Hepatol. 2023. PMID: 36646909 Review.
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. Among authors: unzu c. Hum Gene Ther. 2013 Dec;24(12):1007-17. doi: 10.1089/hum.2013.166. Hum Gene Ther. 2013. PMID: 24070415
24 results