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Alternative options for DNA-based experimental therapy of β-thalassemia

Pages 443-462 | Published online: 13 Mar 2012

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Alessia Finotti, Monica Borgatti, Nicoletta Bianchi, Cristina Zuccato, Ilaria Lampronti & Roberto Gambari. (2016) Orphan Drugs and Potential Novel Approaches for Therapies of β-Thalassemia: Current Status and Future Expectations. Expert Opinion on Orphan Drugs 4:3, pages 299-315.
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Alessia Finotti, Laura Breda, Carsten W Lederer, Nicoletta Bianchi, Cristina Zuccato, Marina Kleanthous, Stefano Rivella & Roberto Gambari. (2015) Recent trends in the gene therapy of β-thalassemia. Journal of Blood Medicine 6, pages 69-85.
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Alessia Finotti & Roberto Gambari. (2014) Recent trends for novel options in experimental biological therapy of β-thalassemia. Expert Opinion on Biological Therapy 14:10, pages 1443-1454.
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Roberto Gambari. (2014) Peptide nucleic acids: a review on recent patents and technology transfer. Expert Opinion on Therapeutic Patents 24:3, pages 267-294.
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Articles from other publishers (17)

Kenneth Lundstrom. (2023) Viral Vectors in Gene Therapy: Where Do We Stand in 2023?. Viruses 15:3, pages 698.
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Ruben Adrian Grosso, Paula Virginia Subirada Caldarone, María Cecilia Sánchez, Gustavo Alberto Chiabrando, María Isabel Colombo & Claudio Marcelo Fader. (2019) Hemin induces autophagy in a leukemic erythroblast cell line through the LRP1 receptor. Bioscience Reports 39:1.
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Samaneh Farashi & Cornelis L. Harteveld. (2018) Molecular basis of α-thalassemia. Blood Cells, Molecules, and Diseases 70, pages 43-53.
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Antreas Afantitis, Georgios Leonis, Roberto Gambari & Georgia Melagraki. (2018) Consensus Predictive Model for Human K562 Cell Growth Inhibition through Enalos Cloud Platform. ChemMedChem 13:6, pages 555-563.
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Giulia Breveglieri, Nicoletta Bianchi, Lucia Carmela Cosenza, Maria Rita Gamberini, Francesco Chiavilli, Cristina Zuccato, Giulia Montagner, Monica Borgatti, Ilaria Lampronti, Alessia Finotti & Roberto Gambari. (2017) An Aγ-globin G->A gene polymorphism associated with β039 thalassemia globin gene and high fetal hemoglobin production. BMC Medical Genetics 18:1.
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Claudio Marcelo Fader, Betiana Nebaí Salassa, Rubén Adrián Grosso, Agustín Nicolás Vergara & María Isabel Colombo. (2016) Hemin induces mitophagy in a leukemic erythroblast cell line. Biology of the Cell 108:4, pages 77-95.
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Nicoletta Bianchi, Lucia Carmela Cosenza, Ilaria Lampronti, Alessia Finotti, Giulia Breveglieri, Cristina Zuccato, Enrica Fabbri, Giovanni Marzaro, Adriana Chilin, Gioia De Angelis, Monica Borgatti, Cristiano Gallucci, Cecilia Alfieri, Michela Ribersani, Antonella Isgr?Marco Marziali, Javid Gaziev, Aldo Morrone, Pietro Sodani, Guido Lucarelli, Roberto Gambari & Katia Paciaroni. (2016) Structural and Functional Insights on an Uncharacterized A?-Globin-Gene Polymorphism Present in Four ?0-Thalassemia Families with High Fetal Hemoglobin Levels. Molecular Diagnosis & Therapy 20:2, pages 161-173.
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Alessia Finotti, Jessica Gasparello, Giulia Breveglieri, Lucia Carmela Cosenza, Giulia Montagner, Alberto Bresciani, Sergio Altamura, Nicoletta Bianchi, Elisa Martini, Eleonora Gallerani, Monica Borgatti & Roberto Gambari. (2015) Development and characterization of K562 cell clones expressing BCL11A-XL: Decreased hemoglobin production with fetal hemoglobin inducers and its rescue with mithramycin. Experimental Hematology 43:12, pages 1062-1071.e3.
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Gerd A. Blobel, David Bodine, Marjorie Brand, John Crispino, Marella F.T.R. de Bruijn, David Nathan, Thalia Papayannopoulou, Catherine Porcher, John Strouboulis, Len Zon, Douglas R. Higgs, George Stamatoyannopoulos & James Douglas Engel. (2015) An international effort to cure a global health problem: A report on the 19th Hemoglobin Switching Conference. Experimental Hematology 43:10, pages 821-837.
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Sachith Mettananda, Richard J. GibbonsDouglas R. Higgs. (2015) α-Globin as a molecular target in the treatment of β-thalassemia. Blood 125:24, pages 3694-3701.
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GIULIA MONTAGNER, CHIARA GEMMO, ENRICA FABBRI, ALEX MANICARDI, IGEA ACCARDO, NICOLETTA BIANCHI, ALESSIA FINOTTI, GIULIA BREVEGLIERI, FRANCESCA SALVATORI, MONICA BORGATTI, ILARIA LAMPRONTI, ALBERTO BRESCIANI, SERGIO ALTAMURA, ROBERTO CORRADINI & ROBERTO GAMBARI. (2015) Peptide nucleic acids targeting β-globin mRNAs selectively inhibit hemoglobin production in murine erythroleukemia cells. International Journal of Molecular Medicine 35:1, pages 51-58.
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Giulia Breveglieri, Irene Mancini, Nicoletta Bianchi, Ilaria Lampronti, Francesca Salvatori, Enrica Fabbri, Cristina Zuccato, Lucia C. Cosenza, Giulia Montagner, Monica Borgatti, Fiorella Altruda, Sharmila Fagoonee, Gianni Carandina, Michele Rubini, Vincenzo Aiello, Laura Breda, Stefano Rivella, Roberto Gambari & Alessia Finotti. (2015) Generation and Characterization of a Transgenic Mouse Carrying a Functional Human β -Globin Gene with the IVSI-6 Thalassemia Mutation . BioMed Research International 2015, pages 1-20.
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Roberto Gambari. (2014) The Role of OMICS Research in Understanding Phenotype Variation in Thalassaemia: The THALAMOSS Project. Thalassemia Reports 4:3, pages 4877.
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Alessia Salvador, Eleonora Brognara, Daniela Vedaldi, Ignazio Castagliuolo, Paola Brun, Cristina Zuccato, Ilaria Lampronti & Roberto Gambari. (2013) Induction of erythroid differentiation and increased globin mRNA production with furocoumarins and their photoproducts. Journal of Photochemistry and Photobiology B: Biology 121, pages 57-66.
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Lefkothea Papadopoulou & Asterios Tsiftsoglou. (2013) The Potential Role of Cell Penetrating Peptides in the Intracellular Delivery of Proteins for Therapy of Erythroid Related Disorders. Pharmaceuticals 6:1, pages 32-53.
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. (2012) Current World Literature. Current Opinion in Pediatrics 24:6, pages 770-779.
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André Katayama Yamada, Rozangela Verlengia & Carlos Roberto Bueno Junior. (2012) Myostatin: genetic variants, therapy and gene doping. Brazilian Journal of Pharmaceutical Sciences 48:3, pages 369-377.
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