New Trends in Personalized Therapy of Thalassemia

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

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