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Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy.
Armbruster N, Lattanzi A, Jeavons M, Van Wittenberghe L, Gjata B, Marais T, Martin S, Vignaud A, Voit T, Mavilio F, Barkats M, Buj-Bello A. Armbruster N, et al. Among authors: lattanzi a. Mol Ther Methods Clin Dev. 2016 Sep 14;3:16060. doi: 10.1038/mtm.2016.60. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27652289 Free PMC article.
Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus.
Antoniani C, Meneghini V, Lattanzi A, Felix T, Romano O, Magrin E, Weber L, Pavani G, El Hoss S, Kurita R, Nakamura Y, Cradick TJ, Lundberg AS, Porteus M, Amendola M, El Nemer W, Cavazzana M, Mavilio F, Miccio A. Antoniani C, et al. Among authors: lattanzi a. Blood. 2018 Apr 26;131(17):1960-1973. doi: 10.1182/blood-2017-10-811505. Epub 2018 Mar 8. Blood. 2018. PMID: 29519807 Free article.
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
Lattanzi A, Camarena J, Lahiri P, Segal H, Srifa W, Vakulskas CA, Frock RL, Kenrick J, Lee C, Talbott N, Skowronski J, Cromer MK, Charlesworth CT, Bak RO, Mantri S, Bao G, DiGiusto D, Tisdale J, Wright JF, Bhatia N, Roncarolo MG, Dever DP, Porteus MH. Lattanzi A, et al. Sci Transl Med. 2021 Jun 16;13(598):eabf2444. doi: 10.1126/scitranslmed.abf2444. Sci Transl Med. 2021. PMID: 34135108 Free PMC article.
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.
Fañanas-Baquero S, Quintana-Bustamante O, Dever DP, Alberquilla O, Sanchez-Dominguez R, Camarena J, Ojeda-Perez I, Dessy-Rodriguez M, Turk R, Schubert MS, Lattanzi A, Xu L, Lopez-Lorenzo JL, Bianchi P, Bueren JA, Behlke MA, Porteus M, Segovia JC. Fañanas-Baquero S, et al. Among authors: lattanzi a. Mol Ther Methods Clin Dev. 2021 May 14;22:237-248. doi: 10.1016/j.omtm.2021.05.001. eCollection 2021 Sep 10. Mol Ther Methods Clin Dev. 2021. PMID: 34485608 Free PMC article.
Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and Krabbe-affected non-human primates by intracerebral lentiviral gene therapy.
Meneghini V, Lattanzi A, Tiradani L, Bravo G, Morena F, Sanvito F, Calabria A, Bringas J, Fisher-Perkins JM, Dufour JP, Baker KC, Doglioni C, Montini E, Bunnell BA, Bankiewicz K, Martino S, Naldini L, Gritti A. Meneghini V, et al. Among authors: lattanzi a. EMBO Mol Med. 2016 May 2;8(5):489-510. doi: 10.15252/emmm.201505850. Print 2016 May. EMBO Mol Med. 2016. PMID: 27025653 Free PMC article.
Population-wide gene disruption in the murine lung epithelium via AAV-mediated delivery of CRISPR-Cas9 components.
Chen H, Durinck S, Patel H, Foreman O, Mesh K, Eastham J, Caothien R, Newman RJ, Roose-Girma M, Darmanis S, Warming S, Lattanzi A, Liang Y, Haley B. Chen H, et al. Among authors: lattanzi a. Mol Ther Methods Clin Dev. 2022 Nov 1;27:431-449. doi: 10.1016/j.omtm.2022.10.016. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36419469 Free PMC article.
151 results