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Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies.
Breda L, Ghiaccio V, Tanaka N, Jarocha D, Ikawa Y, Abdulmalik O, Dong A, Casu C, Raabe TD, Shan X, Danet-Desnoyers GA, Doto AM, Everett J, Bushman FD, Radaelli E, Assenmacher CA, Tarrant JC, Hoepp N, Kurita R, Nakamura Y, Guzikowski V, Smith-Whitley K, Kwiatkowski JL, Rivella S. Breda L, et al. Among authors: casu c. Mol Ther. 2021 Apr 7;29(4):1625-1638. doi: 10.1016/j.ymthe.2020.12.036. Epub 2021 Jan 29. Mol Ther. 2021. PMID: 33515514 Free PMC article.
A preclinical approach for gene therapy of beta-thalassemia.
Breda L, Kleinert DA, Casu C, Casula L, Cartegni L, Fibach E, Mancini I, Giardina PJ, Gambari R, Rivella S. Breda L, et al. Among authors: casu c. Ann N Y Acad Sci. 2010 Aug;1202:134-40. doi: 10.1111/j.1749-6632.2010.05594.x. Ann N Y Acad Sci. 2010. PMID: 20712784 Free PMC article.
Therapeutic hemoglobin levels after gene transfer in β-thalassemia mice and in hematopoietic cells of β-thalassemia and sickle cells disease patients.
Breda L, Casu C, Gardenghi S, Bianchi N, Cartegni L, Narla M, Yazdanbakhsh K, Musso M, Manwani D, Little J, Gardner LB, Kleinert DA, Prus E, Fibach E, Grady RW, Giardina PJ, Gambari R, Rivella S. Breda L, et al. Among authors: casu c. PLoS One. 2012;7(3):e32345. doi: 10.1371/journal.pone.0032345. Epub 2012 Mar 27. PLoS One. 2012. PMID: 22479321 Free PMC article.
Hepcidin agonists as therapeutic tools.
Casu C, Nemeth E, Rivella S. Casu C, et al. Blood. 2018 Apr 19;131(16):1790-1794. doi: 10.1182/blood-2017-11-737411. Epub 2018 Mar 9. Blood. 2018. PMID: 29523504 Free PMC article. Review.
Iron age: novel targets for iron overload.
Casu C, Rivella S. Casu C, et al. Hematology Am Soc Hematol Educ Program. 2014 Dec 5;2014(1):216-21. doi: 10.1182/asheducation-2014.1.216. Epub 2014 Nov 18. Hematology Am Soc Hematol Educ Program. 2014. PMID: 25696858 Free PMC article. Review.
2'-O-methoxyethyl splice-switching oligos correct splicing from IVS2-745 β-thalassemia patient cells restoring HbA production and chain rebalance.
Dong A, Ghiaccio V, Motta I, Guo S, Peralta R, Freier SM, Watt A, Damle S, Ikawa Y, Jarocha D, Chappell M, Stephanou C, Delbini P, Chen C, Christou S, Kleanthous M, Smith-Whitley K, Manwani D, Casu C, Abdulmalik O, Cappellini MD, Rivella S, Breda L. Dong A, et al. Among authors: casu c. Haematologica. 2019 May 1;106(5):1433-1442. doi: 10.3324/haematol.2019.226852. Haematologica. 2019. PMID: 32439726 Free PMC article.
76 results