Today is World Thalassaemia Day – and experts say cures based on gene therapy look increasingly promising.
Both stem cell-based gene therapy and CRISPR technology – aimed at removing faulty DNA – show promise, according to Dr Rabi Hanna, a specialist at the Cleveland Clinic, Ohio, USA.
Dr Hanna said: “Gene therapy has held out the prospect of a cure for several years, with great steps forward being made in basic science, and now we are seeing the first research projects in this field emerge as workable treatments.
“Even if we reduce the number of transfusions needed to maintain a healthy count of red blood cells, that is a positive outcome for the patient. The idea that we have several new treatments on the horizon, not just one, will hopefully give us alternatives for cases where one approach is ineffective.”
Dr Hanna added: “In recent months we have seen positive research data published from trials in human subjects, where gene therapy is working, where the results are lasting, and where feared side-effects are not materialising.
“I am hopeful that we can realistically look forward to seeing these move into a treatment setting in the foreseeable future.”
He went on: “It will take a little more time for these advances to become available as treatments, but we are clearly at the point where breakthroughs in research are translating into breakthroughs in treatment, and that offers real hope to many of the patients suffering from thalassaemia today.”
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