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Journal of Blood Medicine Volume 6, 2015 - Issue
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Review

Recent trends in the gene therapy of β-thalassemia

Alessia Finotti1 Laboratory for the Development of Gene and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Centre of Ferrara University, Ferrara, Italy;2 Associazione Veneta per la Lotta alla Talassemia, Rovigo, Italy;3 Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, Ferrara, Italy
,
Laura Breda4 Department of Pediatrics, Division of Haematology/Oncology, Weill Cornell Medical College, New York, NY, USA
,
Carsten W Lederer6 Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;7 Cyprus School of Molecular Medicine, Nicosia, Cyprus
,
Nicoletta Bianchi1 Laboratory for the Development of Gene and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Centre of Ferrara University, Ferrara, Italy;2 Associazione Veneta per la Lotta alla Talassemia, Rovigo, Italy;3 Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, Ferrara, Italy
,
Cristina Zuccato1 Laboratory for the Development of Gene and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Centre of Ferrara University, Ferrara, Italy;2 Associazione Veneta per la Lotta alla Talassemia, Rovigo, Italy;3 Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, Ferrara, Italy
,
Marina Kleanthous6 Department of Molecular Genetics Thalassaemia, The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus;7 Cyprus School of Molecular Medicine, Nicosia, Cyprus
,
Stefano Rivella4 Department of Pediatrics, Division of Haematology/Oncology, Weill Cornell Medical College, New York, NY, USA;5 Department of Cell and Development Biology, Weill Cornell Medical College, New York, NY, USA
&
Roberto Gambari1 Laboratory for the Development of Gene and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Centre of Ferrara University, Ferrara, Italy;2 Associazione Veneta per la Lotta alla Talassemia, Rovigo, Italy;3 Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Ferrara University, Ferrara, ItalyCorrespondence[email protected]
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Pages 69-85 | Published online: 19 Feb 2015

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Orapan Sripichai & Suthat Fucharoen. (2016) Fetal hemoglobin regulation in β-thalassemia: heterogeneity, modifiers and therapeutic approaches. Expert Review of Hematology 9:12, pages 1129-1137.
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Bruno Deltreggia Benites, Stephany Oliveira Bastos, Gabriel Baldanzi, Allan de Oliveira dos Santos, Celso Dario Ramos, Fernando Ferreira Costa, Simone Cristina Olenscki Gilli & Sara Teresinha Olalla Saad. (2016) Sickle cell/β-thalassemia: Comparison of Sβ0 and Sβ+ Brazilian patients followed at a single institution. Hematology 21:10, pages 623-629.
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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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Raffaella Origa, Alessandro Baldan, Maria Marsella & Caterina Borgna-Pignatti. (2015) A complicated disease: what can be done to manage thalassemia major more effectively?. Expert Review of Hematology 8:6, pages 851-862.
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Alexander J. Grooms, Benjamin J. Burris & Abraham K. Badu‐Tawiah. (2022) Mass spectrometry for metabolomics analysis: Applications in neonatal and cancer screening. Mass Spectrometry Reviews.
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Andrés Lamsfus-CalleAlberto Daniel-MorenoGuillermo Ureña-BailénJennifer RottenbergerJanani RajuThomas EptingSabina MarcianoLukas HeumosPraveen BaskaranJustin S AntonyRupert HandgretingerMarkus Mezger. (2021) Universal Gene Correction Approaches for β-hemoglobinopathies Using CRISPR-Cas9 and Adeno-Associated Virus Serotype 6 Donor Templates. The CRISPR Journal 4:2, pages 207-222.
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E. R. Susanto, A. Syarif, K Muludi, R. R. W. Perdani & A. Wantoro. (2021) Implementation of Fuzzy-based Model for Prediction of Thalassemia Diseases. Journal of Physics: Conference Series 1751:1, pages 012034.
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Andrés Lamsfus-Calle, Alberto Daniel-Moreno, Justin S. Antony, Thomas Epting, Lukas Heumos, Praveen Baskaran, Jakob Admard, Nicolas Casadei, Ngadhnjim Latifi, Darina M. Siegmund, Michael S. D. Kormann, Rupert Handgretinger & Markus Mezger. (2020) Comparative targeting analysis of KLF1, BCL11A, and HBG1/2 in CD34+ HSPCs by CRISPR/Cas9 for the induction of fetal hemoglobin. Scientific Reports 10:1.
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Navid Shomali, Tohid Gharibi, Ghasem Vahedi, Rebar N. Mohammed, Hamed Mohammadi, Sevda Salimifard & Faroogh Marofi. (2019) Mesenchymal stem cells as carrier of the therapeutic agent in the gene therapy of blood disorders. Journal of Cellular Physiology 235:5, pages 4120-4134.
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Mohsen Moghadami, Masoud Sheybani, Abbas Reziaean Zadeh & Tayebeh Rakhshani. (2019) Evaluation of the Prevalence of Thalassemia and Sickle Hemoglobin in Marriage Applicants Referring to the Genetic Disease Counseling Center in Kazeroun, Iran during 2014-2018. Journal of Human, Environment, and Health Promotion 5:4, pages 183-187.
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Lefkothea C. Papadopoulou, Alexandra Ingendoh-Tsakmakidis, Christina N. Mpoutoureli, Lamprini D. Tzikalou, Efthymia D. Spyridou, George I. Gavriilidis, Georgios C. Kaiafas, Agoritsa T. Ntaska, Efthymia Vlachaki, George Panayotou, Martina Samiotaki & Asterios S. Tsiftsoglou. (2018) Production and Transduction of a Human Recombinant β-Globin Chain into Proerythroid K-562 Cells To Replace Missing Endogenous β-Globin. Molecular Pharmaceutics 15:12, pages 5665-5677.
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Özgür Aldemir. 2018. Thalassemia and Other Hemolytic Anemias. Thalassemia and Other Hemolytic Anemias.
Jessica Gasparello, Enrica Fabbri, Nicoletta Bianchi, Giulia Breveglieri, Cristina Zuccato, Monica Borgatti, Roberto Gambari & Alessia Finotti. (2017) BCL11A mRNA Targeting by miR-210: A Possible Network Regulating γ-Globin Gene Expression. International Journal of Molecular Sciences 18:12, pages 2530.
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Paola Spitalieri, Valentina Rosa Talarico, Michela Murdocca, Giuseppe Novelli & Federica Sangiuolo. (2016) Human induced pluripotent stem cells for monogenic disease modelling and therapy. World Journal of Stem Cells 8:4, pages 118.
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Yan-Ling Cheng, Xin-Hua Zhang, Yu-Wen Sun, Wen-Juan Wang, Su-Ping Fang & Zhi-Kui Wu. (2016) Clinical Effect and Mechanism of Yisui Shengxue Granules in Thalassemia Patients with Mild, Moderate, or Severe Anemia. Evidence-Based Complementary and Alternative Medicine 2016, pages 1-13.
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Ravi BirlaRavi Birla. 2016. Tissue Engineering for the Heart. Tissue Engineering for the Heart 1 30 .
Astrid Glaser, Bradley McColl & Jim Vadolas. (2015) The therapeutic potential of genome editing for β-thalassemia. F1000Research 4, pages 1431.
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