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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. PLoS One. 2012;7(3):e32345. doi: 10.1371/journal.pone.0032345. Epub 2012 Mar 27. PLoS One. 2012. PMID: 22479321 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. 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.