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Special Issue "New Trends in Personalized Therapy of Thalassemia"

A special issue of Journal of Clinical Medicine (ISSN 2077-0383). This special issue belongs to the section "Hematology".

Deadline for manuscript submissions: 30 June 2019

Special Issue Editors

Guest Editor
Dr. Roberto Gambari

Department of Life Sciences and Biotechnology, Section of Biochemistry and Molecular Biology, Via Fossato di Mortara, 74, 44100 Ferrara, Italy
Website | E-Mail
Phone: +39-0532-974443
Fax: +39-0532-974500
Interests: Patients stratification; OMICS analyses; Transcriptional regulation of globin genes; Gene therapy; Induction of Fetal Hemoglobin; MicroRNAs; In vitro experimental model systems; Transgenic animals; Molecular diagnosis
Guest Editor
Dr. Eitan Fibach

Department of Hematology, Hadassah - Hebrew University Medical Center, Ein-Kerem, Jerusalem, Israel
Website | E-Mail
Phone: 972-2-6776751
Fax: 972-2-6423067
Interests: Gene therapy; Induction of Fetal Hemoglobin; In vitro experimental model systems; Molecular diagnosis

Special Issue Information

Dear Colleagues,

In beta−thalassemias, mutations of the beta-globin gene or its regulatory regions cause the absence or reduced synthesis of beta-globin chains. The disease is associated with morbidity and mortality due to severe chronic anemia or treatment-related complications. In the last years novel promising strategies have been developed for implementing therapeutic and diagnostic protocols regarding beta-thalassemia and related diseases (such as sickle cell anemia).

For instance, the first trials on gene therapy have been recently reported, based on the use of new generation of lentiviral vectors; induction of fetal hemoglobin using hydroxyurea is now firmly established as a strategy to ameliorate the clinical parameters of beta-thalassemia patients. These achievements are based on the development of in vitro and in vivo experimental systems, on the design on novel drugs (including DNA-based molecules) and suitable delivery systems for personalized therapy. Future developments are expected in the field of stem cell therapy, including induced pluripotent stem cells (iPS) derived from thalassemia patients. Diagnostic approaches are expected to bring important achievements in the field of non-invasive prenatal diagnosis.

We are particularly interested in review and research manuscripts that report the relevance of novel approaches for gene therapy, gene editing and improved diagnosis, prognosis and management of beta-thalassemias. Reviews that summarize the results of clinical trials are welcome as well. Moreover, papers dealing with methodological achievements leading to the development of novel therapeutic and diagnostic protocols facilitating personalized therapy in precision medicine would be of great interest.

Main topics include, but are not limited to:

  • Patients stratification
  • OMICS analyses
  • Transcriptional regulation of globin genes
  • Gene therapy for thalassemia
  • Gene editing
  • Read-through approaches for stop-codon mutations
  • Induction of Fetal Hemoglobin
  • Fetal globin modifiers
  • Stem cells
  • Development of iPS
  • MicroRNAs
  • Long non-coding RNAs
  • In vitro experimental model systems
  • In vivo experimental model systems, including transgenic animals
  • Molecular diagnosis
  • Prenatal diagnosis

Dr. Roberto Gambari
Dr. Eitan Fibach
Guest Editors

Manuscript Submission Information

Manuscripts should be submitted online at www.mdpi.com by registering and logging in to this website. Once you are registered, click here to go to the submission form. Manuscripts can be submitted until the deadline. All papers will be peer-reviewed. Accepted papers will be published continuously in the journal (as soon as accepted) and will be listed together on the special issue website. Research articles, review articles as well as short communications are invited. For planned papers, a title and short abstract (about 100 words) can be sent to the Editorial Office for announcement on this website.

Submitted manuscripts should not have been published previously, nor be under consideration for publication elsewhere (except conference proceedings papers). All manuscripts are thoroughly refereed through a single-blind peer-review process. A guide for authors and other relevant information for submission of manuscripts is available on the Instructions for Authors page. Journal of Clinical Medicine is an international peer-reviewed open access monthly journal published by MDPI.

Please visit the Instructions for Authors page before submitting a manuscript. The Article Processing Charge (APC) for publication in this open access journal is 1800 CHF (Swiss Francs). Submitted papers should be well formatted and use good English. Authors may use MDPI's English editing service prior to publication or during author revisions.


  • Thalassemia
  • Theranostics
  • Patients stratification
  • Transcriptional regulation
  • Gene therapy
  • Gene editing
  • Read-through approaches
  • Induction of Fetal Hemoglobin
  • MicroRNAs
  • Prenatal diagnosis
  • Transgenic animals

Published Papers

This special issue is now open for submission.
J. Clin. Med. EISSN 2077-0383 Published by MDPI AG, Basel, Switzerland RSS E-Mail Table of Contents Alert
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