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Expert Review of Hematology Volume 6, 2013 - Issue 3
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Combining gene therapy and fetal hemoglobin induction for treatment of β-thalassemia

Laura Breda Department of Pediatrics, Division of Hematology–Oncology, Weill Cornell Medical College, New York, NY, USA. [email protected]; Department of Pediatrics, Division of Hematology–Oncology, Weill Cornell Medical College, New York, NY, USA. [email protected]
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Stefano Rivella Department of Pediatrics, Division of Hematology–Oncology, Weill Cornell Medical College, New York, NY, USA. [email protected]; Department of Cell & Development Biology, Weill Cornell Medical College, New York, NY, USA; Department of Pediatrics, Division of Hematology–Oncology, Weill Cornell Medical College, New York, NY, USA. [email protected]; Department of Cell & Development Biology, Weill Cornell Medical College, New York, NY, USA
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Cristina Zuccato Department of Life Sciences & Biotechnology, University of Ferrara, Ferrara, Italy; Department of Life Sciences & Biotechnology, University of Ferrara, Ferrara, Italy
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Roberto Gambari Department of Life Sciences & Biotechnology, University of Ferrara, Ferrara, Italy; Laboratory for the Development of Gene and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Centre of Ferrara University, Ferrara, Italy; Department of Life Sciences & Biotechnology, University of Ferrara, Ferrara, Italy; Laboratory for the Development of Gene and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Centre of Ferrara University, Ferrara, Italy
Pages 255-264 | Published online: 10 Jan 2014

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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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Articles from other publishers (10)

Alessia Finotti & Roberto Gambari. (2023) Combined approaches for increasing fetal hemoglobin (HbF) and de novo production of adult hemoglobin (HbA) in erythroid cells from β-thalassemia patients: treatment with HbF inducers and CRISPR-Cas9 based genome editing. Frontiers in Genome Editing 5.
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Lucia Carmela Cosenza, Cristina Zuccato, Matteo Zurlo, Roberto Gambari & Alessia Finotti. (2022) Co-Treatment of Erythroid Cells from β-Thalassemia Patients with CRISPR-Cas9-Based β039-Globin Gene Editing and Induction of Fetal Hemoglobin. Genes 13:10, pages 1727.
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Joachim B. Kunz & Andreas E. Kulozik. (2020) Gene Therapy of the Hemoglobinopathies. HemaSphere 4:5, pages e479.
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Valentina Ghiaccio, Maxwell Chappell, Stefano Rivella & Laura Breda. (2019) Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges. Molecular Diagnosis & Therapy 23:2, pages 173-186.
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Sandeep Soni. (2017) Novel and innovative approaches for treatment of β-thalassemia. Pediatric Hematology Oncology Journal 2:4, pages 121-126.
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Matthew C. CanverStuart H. Orkin. (2016) Customizing the genome as therapy for the β-hemoglobinopathies. Blood 127:21, pages 2536-2545.
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Michael A Goodman & Punam Malik. 2005. Encyclopedia of Life Sciences. Encyclopedia of Life Sciences 1 8 .
. (2015) Thalassemia Modular Stratification System for Personalized Therapy of Beta-Thalassemia (THALAMOSS). Human Gene Therapy Clinical Development 26:2, pages 100-102.
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Eleni Papanikolaou, Anna Paruzynski, Ioannis Kasampalidis, Annette Deichmann, Evangelos Stamateris, Manfred Schmidt, Christof von Kalle & Nicholas P Anagnou. (2015) Cell Cycle Status of CD34+ Hemopoietic Stem Cells Determines Lentiviral Integration in Actively Transcribed and Development-related Genes. Molecular Therapy 23:4, pages 683-696.
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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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