Introduction: The β-thalassemias are rare diseases caused by more than 300 different mutations of the adult β-globin genes: they are treated with blood transfusions, chelation therapy or bone marrow transplantation.Areas Covered: This article reviews orphan drugs, related patent applications and related clinical trials on β-thalassemia, including novel therapeutic approaches and repurposing of orphan drugs.Expert Opinion: Patient stratification is expected to be applied for personalized therapeutic interventions for β-thalassemia patients. This will cover primary mutations of the β-globin gene and DNA polymorphisms that predict severity of the disease and response to therapy. Improved understanding of the regulation of γ-globin gene expression and the optimization of novel approaches for gene editing are expected to introduce innovative orphan drugs and/or approaches for fetal hemoglobin induction and gene correction, respectively. The possible combination between gene therapy and cellular therapy, including gene-correction, presents a new opportunity. A dramatic increase in the need for cellular biobanking (including also cord blood biobanking) is expected, given the possibility to intervene on patients-derived cells with bone marrow transplantation and/or generated induced pluripotent stem cells. The repurposing of drugs used in clinical trials for different pathologies is an interesting approach to providing novel drugs at low cost.
Scheda prodotto non validato
Attenzione! I dati visualizzati non sono stati sottoposti a validazione da parte dell'ateneo
|Titolo:||Orphan Drugs and Potential Novel Approaches for Therapies of β-Thalassemia: Current Status and Future Expectations|
|Data di pubblicazione:||2016|
|Appare nelle tipologie:||03.1 Articolo su rivista|