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Current clinical management for these patients depends on repeated transfusions followed by iron\u2010chelating therapy. Several novel approaches to correct the resulting \u03b1\/\u03b2\u2010globin chain imbalance, treat ineffective erythropoiesis and improve iron overload are currently being developed. Up to now, the only curative treatment for \u03b2\u2010thalassemia is haematopoietic stem\u2010cell transplantation, but this is a risky and costly procedure. Gene therapy, gene editing and base editing are emerging as a powerful approach to treat this disease. In \u03b2\u2010thalassaemia, gene therapy involves the insertion of a vector containing the normal \u03b2\u2010globin or \u03b3\u2010globin gene into haematopoietic stem cells to permanently produce normal red blood cells. Gene editing and base editing involves the use of zinc finger nucleases, transcription activator\u2010like nucleases and clustered regularly interspaced short palindromic repeats\/Cas9 to either correct the causative mutation or else insert a single nucleotide variant that will increase foetal haemoglobin. In this review, we will examine the current management strategies used to treat \u03b2\u2010thalassaemia and focus on the novel therapies targeting ineffective erythropoiesis, improving iron overload and correction of the globin chain imbalance.<\/jats:p>","DOI":"10.1111\/bcp.14918","type":"journal-article","created":{"date-parts":[[2021,5,18]],"date-time":"2021-05-18T17:02:15Z","timestamp":1621357335000},"page":"2509-2524","source":"Crossref","is-referenced-by-count":15,"title":["Novel therapies in \u03b2\u2010thalassaemia"],"prefix":"10.1111","volume":"88","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1661-7158","authenticated-orcid":false,"given":"Laura","family":"Grech","sequence":"first","affiliation":[{"name":"Centre for Molecular Medicine and Biobanking University of Malta  Malta"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karen","family":"Borg","sequence":"additional","affiliation":[{"name":"Department of Public Health Medicine Ministry for Health  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