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Gene therapy approach for haemoglobinopathies
Researchers have demonstrated the ability to induce production of fetal haemoglobin in adult blood cells, which could potentially be used in developing new therapies for sickle cell disease and other haemoglobinopathies.
The scientists used a technique called forced chromatin looping to effectively reprogramme cellular gene expression, switching cells to production of the form of haemoglobin that normally disappears after birth. Reactivation of this functional fetal haemoglobin could be an effective treatment for patients with dysfunctional mutations in their adult haemoglobin genes, notably sickle cell anaemia and beta-thalassaemia.
The switch was achieved using a genetically engineered DNA binding motif called a zinc finger targeted to the fetal haemoglobin gene. The Children's Hospital of Philadelphia researcher Dr Jeremy Rupon reportedly said: "If we can translate this approach to humans, we may enable new treatment options for patients".
The results were presented at a press conference during the annual meeting of the American Society of Hematology.