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mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks.
Jericó D, Córdoba KM, Jiang L, Schmitt C, Morán M, Sampedro A, Alegre M, Collantes M, Santamaría E, Alegre E, Culerier C, de Mendoza AE, Oyarzabal J, Martín MA, Peñuelas I, Ávila MA, Gouya L, Martini PGV, Fontanellas A. Jericó D, et al. Among authors: sampedro a. Mol Ther Nucleic Acids. 2021 May 19;25:207-219. doi: 10.1016/j.omtn.2021.05.010. eCollection 2021 Sep 3. Mol Ther Nucleic Acids. 2021. PMID: 34458006 Free PMC article.
Epidermal growth factor receptor ligands in murine models for erythropoietic protoporphyria: potential novel players in the progression of liver injury.
Berasain C, Sampedro A, Mauleón I, Goñi S, Latasa MU, Matscheko N, García-Bravo M, Unzu C, Corrales FJ, Enríquez de Salamanca R, Prieto J, Avila MA, Fontanellas A. Berasain C, et al. Among authors: sampedro a. Cell Mol Biol (Noisy-le-grand). 2009 Feb 16;55(1):29-37. Cell Mol Biol (Noisy-le-grand). 2009. PMID: 19267999
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. Among authors: sampedro a. 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.
Intensive pharmacological immunosuppression allows for repetitive liver gene transfer with recombinant adenovirus in nonhuman primates.
Fontanellas A, Hervás-Stubbs S, Mauleón I, Dubrot J, Mancheño U, Collantes M, Sampedro A, Unzu C, Alfaro C, Palazón A, Smerdou C, Benito A, Prieto J, Peñuelas I, Melero I. Fontanellas A, et al. Among authors: sampedro a. Mol Ther. 2010 Apr;18(4):754-65. doi: 10.1038/mt.2009.312. Epub 2010 Jan 19. Mol Ther. 2010. PMID: 20087317 Free PMC article.
Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.
Unzu C, Sampedro A, Mauleón I, Alegre M, Beattie SG, de Salamanca RE, Snapper J, Twisk J, Petry H, González-Aseguinolaza G, Artieda J, Rodríguez-Pena MS, Prieto J, Fontanellas A. Unzu C, et al. Among authors: sampedro a. Mol Ther. 2011 Feb;19(2):243-50. doi: 10.1038/mt.2010.210. Epub 2010 Sep 28. Mol Ther. 2011. PMID: 20877347 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. Among authors: sampedro a. Hum Gene Ther. 2013 Dec;24(12):1007-17. doi: 10.1089/hum.2013.166. Hum Gene Ther. 2013. PMID: 24070415
196 results