title: Recent trends for novel options in experimental biological therapy of β-thalassemia.
abstract: BACKGROUND
  β-thalassemias are caused by nearly 300 mutations of the β-globin gene, leading to low or absent production of adult hemoglobin. Achievements have been recently obtained on innovative therapeutic strategies for β-thalassemias, based on studies focusing on the transcriptional regulation of the γ-globin genes, epigenetic mechanisms governing erythroid differentiation, gene therapy and genetic correction of the mutations.
  
  
  METHODS
  The objective of this review is to describe recently published approaches (the review covers the years 2011 - 2014) useful for the development of novel therapeutic strategies for the treatment of β-thalassemia.
  
  
  CONCLUSIONS
  Modification of β-globin gene expression in β-thalassemia cells was achieved by gene therapy (eventually in combination with induction of fetal hemoglobin [HbF]) and correction of the mutated β-globin gene. Based on recent areas of progress in understanding the control of γ-globin gene expression, novel strategies for inducing HbF have been proposed. Furthermore, the identification of microRNAs involved in erythroid differentiation and HbF production opens novel options for developing therapeutic approaches for β-thalassemia and sickle-cell anemia.
authors.affiliation: Biotechnology Centre of Ferrara University, Laboratory for the Development of Gene and Pharmacogenomic Therapy of Thalassaemia , Ferrara , Italy.
authors.forename: Alessia
authors.initials: A
authors.lastname: Finotti
authors.forename: Roberto
authors.initials: R
authors.lastname: Gambari
mesh_terms: D001691:Biological Therapy / Q000379:methods*; D044127:Epigenesis, Genetic; D005786:Gene Expression Regulation / Q000502:physiology*; D015316:Genetic Therapy / Q000379:methods; D006801:Humans; D055544:beta-Globins / Q000235:genetics*; D017086:beta-Thalassemia / Q000235:genetics / Q000628:therapy*
chemical_list: D055544:beta-Globins
keywords: fetal hemoglobin induction; gene therapy; induced pluripotent cells; microRNA; thalassemia; transcription factors