Open access peer-reviewed Edited Volume

Beta Thalassemia

Marwa Zakaria

Zagazig University


Tamer Hassan

Zagazig University


unbalanced alpha chain ineffective erythropioesis genetic modifiers molecular basis wild mutations benign mutations thalasemia major thalasemia intermedia endocrinal complications preimplantation genetic diagnosis cardiac t2* LIC SQUID stem cell transplantation gene therapy iron metabolism manipulators

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About the book

Beta-thalassemia is classified into three main subgroups based on their clinical expression: major, intermedia, and minor. β-thalassemia major presents itself within the first 2 years of life with severe anemia, poor growth, and skeletal abnormalities and requires regular, lifelong blood transfusions. Thanks to the significant improvement in therapy, patients with β-thalassemia may reach an advanced age. This is associated with clinical symptoms that are the consequence of the disease itself and the treatment modalities. Agents such as JAK2 inhibitors and TGF-β ligand traps that reduce the ineffective erythropoiesis process are already being tested in clinical trials with promising results. Other agents that aim to reduce oxidative stress (activators of Foxo3, HRI-eIF2aP, Prx2, Hsp70, and PK anti-oxidant systems and inhibitors of HO-1) and to decrease iron overload (hepcidin agonists, erythroferrone inhibitors and exogenous transferrin) are also under experimental investigation. Significant progress has also been made in the area of allogeneic hematopoietic stem cell transplantation with several ongoing clinical trials examining new condition regimens as well as different donor selection and stem cell source options. Gene therapy has reached a critical point and phase 1 clinical trials have recently been launched to examine the effectiveness and especially long term safety. Epigenetic manipulation and genomic editing of the γ- or β-globin gene are novel and promising experimental gene therapy approaches for β-thalassemia giving hope for cure for this chronic disease.
This book will shed the light on recent findings regarding the pathophysiology underlying the major symptoms of β-thalassemia and potential therapeutic modalities for the amelioration of its complications, as well as new modalities that may provide a cure for the disease, also we will outlines the key points of the molecular mechanisms underlying β-thalassemia in relation to the development of new therapies and an update is given both at the pre-clinical and clinical level.

Publishing process

Book initiated and editor appointed

Date completed: August 27th 2019

Applications to edit the book are assessed and a suitable editor is selected, at which point the process begins.

Chapter proposals submitted and reviewed

Deadline Extended: Open for Submissions

Potential authors submit chapter proposals ready for review by the academic editor and our publishing review team.

Approved chapters written in full and submitted

Deadline for full chapters: November 16th 2019

Once approved by the academic editor and publishing review team, chapters are written and submitted according to pre-agreed parameters

Full chapters peer reviewed

Review results due: February 4th 2020

Full chapter manuscripts are screened for plagiarism and undergo a Main Editor Peer Review. Results are sent to authors within 30 days of submission, with suggestions for rounds of revisions.

Book compiled, published and promoted

Expected publication date: April 4th 2020

All chapters are copy-checked and typesetted before being published. IntechOpen regularly submits its books to major databases for evaluation and coverage, including the Clarivate Analytics Book Citation Index in the Web of ScienceTM Core Collection. Other discipline-specific databases are also targeted, such as Web of Science's BIOSIS Previews.

About the editor

Marwa Zakaria

Zagazig University

Assistant Professor of Pediatrics and pediatric Hematology and Oncology, Pediatric Department, Zagazig University, Egypt. Active member in SIOP, EHA, HAA. Professional training and workshops including; ICH GCP. EHA-master class 2015-2016 and EHA- Bite size master Class 2017-2018, Training from Wilkins-Barrick Society of Neurooncology (SNO) Marrakesh, Morocco, Completion of Pediatric Nutrition course (School of Medicine Boston University 2017). International scholarship and Preceptorship including: Thalassemia (Beirut – Lebanon September 2015). SIOP scholarship 2018. EHA-HOPE scholarship Cairo 2017 and 2018. Guest speaker at numerous international pediatric oncology and hematology meetings. Had over 20 international research publications in The field of Pediatrics and Pediatric Hematology & Oncology. Academic editor of online book and author of two online book chapters. Reviewer in international and national peer reviewed journals (Medicine (Baltimore) Intech Open Publisher, Frontiers in Pediatrics, Molecular Medicine Reports, Zagazig University medical journal. Sub-investigator in 6 international Clinical trials.

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