Review

GENE MANIPULATION

Franco Locatelli^{1

2}, Mattia Algeri^{1

3}

A.T. Taher¹, D. Farmakis², J.B. Porter³, M.D. Cappellini⁴, K.M. Musallam⁵

, editors.

In: Guidelines for the Management of Transfusion-Dependent β-Thalassaemia (TDT) [Internet]. 5th edition. Nicosia, Cyprus: Thalassaemia International Federation; 2025. Chapter 15.

Affiliations

¹ Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children’s Hospital, Rome, Italy
² Catholic University of the Sacred Heart, Rome, Italy
³ Department of Health Sciences, Magna Graecia University, Catanzaro, Italy

Book Affiliations

¹ Professor of Internal Medicine, Hematology & Oncology; Associate VP for Academic Centers, Development and External Affairs; Director, Naef K. Basile Cancer Institute, American University of Beirut Medical Center, Beirut, Lebanon
² Associate Professor, Department of Cardiology, Athens University Hospital Attikon, National and Kapodistrian University of Athens Medical School, Athens, Greece
³ Professor of Haematology (Emeritus) and Consultant Haematologist, University College London Hospitals, London, United Kingdom
⁴ Professor of Medicine, Unit of Medicine and Metabolic Disease, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
⁵ Group Chief Research Officer (Burjeel Holdings) and Founding Director, Center for Research on Rare Blood Disorders (CR-RBD), Burjeel Medical City, Abu Dhabi, United Arab Emirates; Adjunct Professor, Department of Public Health & Epidemiology, Khalifa University, Abu Dhabi, United Arab Emirates; Adjunct Professor, Division of Hematology/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA

PMID: 40367262
Bookshelf ID: NBK614241

Free Books & Documents

Review

GENE MANIPULATION

Franco Locatelli et al.

Free Books & Documents

In: Guidelines for the Management of Transfusion-Dependent β-Thalassaemia (TDT) [Internet]. 5th edition. Nicosia, Cyprus: Thalassaemia International Federation; 2025. Chapter 15.

Authors

Franco Locatelli^{1

2}, Mattia Algeri^{1

3}

Book Editors

A.T. Taher¹, D. Farmakis², J.B. Porter³, M.D. Cappellini⁴, K.M. Musallam⁵

Affiliations

¹ Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, IRCCS Bambino Gesu Children’s Hospital, Rome, Italy
² Catholic University of the Sacred Heart, Rome, Italy
³ Department of Health Sciences, Magna Graecia University, Catanzaro, Italy

Book Affiliations

¹ Professor of Internal Medicine, Hematology & Oncology; Associate VP for Academic Centers, Development and External Affairs; Director, Naef K. Basile Cancer Institute, American University of Beirut Medical Center, Beirut, Lebanon
² Associate Professor, Department of Cardiology, Athens University Hospital Attikon, National and Kapodistrian University of Athens Medical School, Athens, Greece
³ Professor of Haematology (Emeritus) and Consultant Haematologist, University College London Hospitals, London, United Kingdom
⁴ Professor of Medicine, Unit of Medicine and Metabolic Disease, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy
⁵ Group Chief Research Officer (Burjeel Holdings) and Founding Director, Center for Research on Rare Blood Disorders (CR-RBD), Burjeel Medical City, Abu Dhabi, United Arab Emirates; Adjunct Professor, Department of Public Health & Epidemiology, Khalifa University, Abu Dhabi, United Arab Emirates; Adjunct Professor, Division of Hematology/Oncology, Department of Pediatrics, Weill Cornell Medicine, New York, NY, USA

PMID: 40367262
Bookshelf ID: NBK614241

No abstract available

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Conflict of interest statement

FL reports participating in speaker’s bureau with Amgen, Bristol Myers Squibb, Miltenyi Biotec, Neovii, Novartis, SOBI and Vertex Pharmaceuticals; and consultancy for Sanofi, Vertex Pharmaceuticals and Amgen; and honoraria, advisory board participation for Amgen. MA reports scientific advisory board and steering committee participation for Vertex Pharmaceuticals. All competing interests are outside the present work.

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References

1. Magrin E, Miccio A, Cavazzana M Lentiviral and genome-editing strategies for the treatment of betahemoglobinopathies. Blood. 2019;134(15):1203–1213. - PubMed
1. Algeri M, Lodi M, Locatelli F Hematopoietic Stem Cell Transplantation in Thalassemia. Hematol Oncol Clin North Am. 2023;37(2):413–432. - PubMed
1. Cline MJ Perspectives for gene therapy: inserting new genetic information into mammalian cells by physical techniques and viral vectors. Pharmacol Ther. 1985;29(1):69–92. - PubMed
1. Bulcha JT, Wang Y, Ma H, Tai PWL, Gao G Viral vector platforms within the gene therapy landscape. Signal Transduct Target Ther. 2021;6(1):53. - PMC - PubMed
1. Ferrari G, Thrasher AJ, Aiuti A Gene therapy using haematopoietic stem and progenitor cells. Nat Rev Genet. 2021;22(4):216–234. - PubMed

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GENE MANIPULATION

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Book Affiliations

GENE MANIPULATION

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Book Affiliations

Conflict of interest statement

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