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Page 1
A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.
Hermans C, Valentino LA, Thornburg CD, Unzu C, Kay MA, Peyvandi F, Smith P, Miesbach W, McKeown W, Pierce GF, Khair K, Pipe SW, Starcevic K, Pillai M, Jones M, Chiao M, Antonino I, Kessler C. Hermans C, et al. Among authors: unzu c. Haemophilia. 2024 Nov;30(6):1272-1280. doi: 10.1111/hae.15108. Epub 2024 Oct 22. Haemophilia. 2024. PMID: 39437171 Free PMC article.
Systemic messenger RNA replacement therapy is effective in a novel clinically relevant model of acute intermittent porphyria developed in non-human primates.
Córdoba KM, Jericó D, Jiang L, Collantes M, Alegre M, García-Ruiz L, Manzanilla O, Sampedro A, Herranz JM, Insausti I, Martinez de la Cuesta A, Urigo F, Alcaide P, Morán M, Martín MA, Lanciego JL, Lefebvre T, Gouya L, Quincoces G, Unzu C, Hervas-Stubbs S, Falcón-Pérez JM, Alegre E, Aldaz A, Fernández-Seara MA, Peñuelas I, Berraondo P, Martini PGV, Avila MA, Fontanellas A. Córdoba KM, et al. Among authors: unzu c. Gut. 2024 Nov 23:gutjnl-2024-332619. doi: 10.1136/gutjnl-2024-332619. Online ahead of print. Gut. 2024. PMID: 39366725 Free article.
Enhanced AAV transduction across preclinical CNS models: A comparative study in human brain organoids with cross-species evaluations.
Drouyer M, Merjane J, Nedelkoska T, Westhaus A, Scott S, Lee S, Burke PGR, McMullan S, Lanciego JL, Vicente AF, Bugallo R, Unzu C, González-Aseguinolaza G, Gonzalez-Cordero A, Lisowski L. Drouyer M, et al. Among authors: unzu c. Mol Ther Nucleic Acids. 2024 Jun 28;35(3):102264. doi: 10.1016/j.omtn.2024.102264. eCollection 2024 Sep 10. Mol Ther Nucleic Acids. 2024. PMID: 39108635 Free PMC article.
Harnessing whole human liver ex situ normothermic perfusion for preclinical AAV vector evaluation.
Cabanes-Creus M, Liao SHY, Gale Navarro R, Knight M, Nazareth D, Lau NS, Ly M, Zhu E, Roca-Pinilla R, Bugallo Delgado R, Vicente AF, Baltazar G, Westhaus A, Merjane J, Crawford M, McCaughan GW, Unzu C, González-Aseguinolaza G, Alexander IE, Pulitano C, Lisowski L. Cabanes-Creus M, et al. Among authors: unzu c. Nat Commun. 2024 Mar 14;15(1):1876. doi: 10.1038/s41467-024-46194-y. Nat Commun. 2024. PMID: 38485924 Free PMC 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.
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.
24 results