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Promoterless Gene Targeting Approach Combined to CRISPR/Cas9 Efficiently Corrects Hemophilia B Phenotype in Neonatal Mice.
Front Genome Ed. 2022 Mar 11;4:785698. doi: 10.3389/fgeed.2022.785698. eCollection 2022.
Front Genome Ed. 2022.
PMID: 35359664
Free PMC article.
Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model.
Porro F, Bortolussi G, Barzel A, De Caneva A, Iaconcig A, Vodret S, Zentilin L, Kay MA, Muro AF.
Porro F, et al. Among authors: de caneva a.
EMBO Mol Med. 2017 Oct;9(10):1346-1355. doi: 10.15252/emmm.201707601.
EMBO Mol Med. 2017.
PMID: 28751579
Free PMC article.
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Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases.
De Caneva A, Porro F, Bortolussi G, Sola R, Lisjak M, Barzel A, Giacca M, Kay MA, Vlahoviček K, Zentilin L, Muro AF.
De Caneva A, et al.
JCI Insight. 2019 Jun 18;5(15):e128863. doi: 10.1172/jci.insight.128863.
JCI Insight. 2019.
PMID: 31211694
Free PMC article.
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Generation of Ugt1-deficient murine liver cell lines using TALEN technology.
Porro F, Bockor L, De Caneva A, Bortolussi G, Muro AF.
Porro F, et al. Among authors: de caneva a.
PLoS One. 2014 Aug 13;9(8):e104816. doi: 10.1371/journal.pone.0104816. eCollection 2014.
PLoS One. 2014.
PMID: 25118822
Free PMC article.
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CRISPR-Cas9-mediated somatic correction of a one-base deletion in the Ugt1a gene ameliorates hyperbilirubinemia in Crigler-Najjar syndrome mice.
Bortolussi G, Iaconcig A, Canarutto G, Porro F, Ferrucci F, Galletta C, Díaz-Muñoz C, Rawat V, De Caneva A, Olajide OJ, Zentilin L, Piazza S, Bočkor L, Muro AF.
Bortolussi G, et al. Among authors: de caneva a.
Mol Ther Methods Clin Dev. 2023 Nov 19;31:101161. doi: 10.1016/j.omtm.2023.101161. eCollection 2023 Dec 14.
Mol Ther Methods Clin Dev. 2023.
PMID: 38094199
Free PMC article.
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