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Progress and harmonization of gene editing to treat human diseases: Proceeding of COST Action CA21113 GenE-HumDi.
Cavazza A, Hendel A, Bak RO, Rio P, Güell M, Lainšček D, Arechavala-Gomeza V, Peng L, Hapil FZ, Harvey J, Ortega FG, Gonzalez-Martinez C, Lederer CW, Mikkelsen K, Gasiunas G, Kalter N, Gonçalves MAFV, Petersen J, Garanto A, Montoliu L, Maresca M, Seemann SE, Gorodkin J, Mazini L, Sanchez R, Rodriguez-Madoz JR, Maldonado-Pérez N, Laura T, Schmueck-Henneresse M, Maccalli C, Grünewald J, Carmona G, Kachamakova-Trojanowska N, Miccio A, Martin F, Turchiano G, Cathomen T, Luo Y, Tsai SQ, Benabdellah K; COST Action CA21113. Cavazza A, et al. Among authors: turchiano g. Mol Ther Nucleic Acids. 2023 Oct 29;34:102066. doi: 10.1016/j.omtn.2023.102066. eCollection 2023 Dec 12. Mol Ther Nucleic Acids. 2023. PMID: 38034032 Free PMC article. Review.
Gene Editing and Genotoxicity: Targeting the Off-Targets.
Blattner G, Cavazza A, Thrasher AJ, Turchiano G. Blattner G, et al. Among authors: turchiano g. Front Genome Ed. 2020 Dec 10;2:613252. doi: 10.3389/fgeed.2020.613252. eCollection 2020. Front Genome Ed. 2020. PMID: 34713236 Free PMC article. Review.
Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells.
Patsali P, Turchiano G, Papasavva P, Romito M, Loucari CC, Stephanou C, Christou S, Sitarou M, Mussolino C, Cornu TI, Antoniou MN, Lederer CW, Cathomen T, Kleanthous M. Patsali P, et al. Among authors: turchiano g. Haematologica. 2019 Nov;104(11):e497-e501. doi: 10.3324/haematol.2018.215178. Epub 2019 Apr 19. Haematologica. 2019. PMID: 31004018 Free PMC article. No abstract available.
Preclinical model for phenotypic correction of dystrophic epidermolysis bullosa by in vivo CRISPR-Cas9 delivery using adenoviral vectors.
García M, Bonafont J, Martínez-Palacios J, Xu R, Turchiano G, Svensson S, Thrasher AJ, Larcher F, Del Rio M, Hernández-Alcoceba R, Garín MI, Mencía Á, Murillas R. García M, et al. Among authors: turchiano g. Mol Ther Methods Clin Dev. 2022 Sep 16;27:96-108. doi: 10.1016/j.omtm.2022.09.005. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36212909 Free PMC article.
Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia.
Bahal S, Zinicola M, Moula SE, Whittaker TE, Schejtman A, Naseem A, Blanco E, Vetharoy W, Hu YT, Rai R, Gomez-Castaneda E, Cunha-Santos C, Burns SO, Morris EC, Booth C, Turchiano G, Cavazza A, Thrasher AJ, Santilli G. Bahal S, et al. Among authors: turchiano g. J Allergy Clin Immunol. 2024 Mar 11:S0091-6749(24)00240-9. doi: 10.1016/j.jaci.2024.03.003. Online ahead of print. J Allergy Clin Immunol. 2024. PMID: 38479630 Free article.
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