London: Italian scientists pioneering a new gene transfer treatment for blood disorder ?-thalassemia have successfully completed preclinical trials, claiming they can correct the lack of beta-globin in patients` blood cells which causes the disease.
The research reveals how gene therapy may represent a safe alternative to current cures that are limited to minority of patients, the `EMBO Molecular Medicine` journal reported.
The disorder-thalassemia is caused when a patient cannot produce enough of ?-globin component of haemoglobin, the protein used by red blood cells to carry oxygen around the body. The lack of-globin causes life threatening anemia, leading to severe damage of the body`s major organs.
"Currently treatments are limited to lifelong regular blood transfusions, and iron chelation to prevent fatal iron overload. The alternative is bone marrow transplantation, an option open to less than 25 per cent of patients.
"Our research has focused on gene therapy – by transplanting genetically corrected stem cells we can restore haemoglobin production and overcome the disorder," said lead scientist Dr Giuliana Ferrari of the San Raffaele Telethon Institute for Gene Therapy in Milan.