Deletions and duplications of single-base pairs typically arise during homologous recombination and cause diseases. But what happens when a mutation occurs over multiple genes?
The cells were transduced with a lentiviral vector delivering the gene encoding the anti-sickling beta globin HbAS3 protein. The investigators showed that lentiviral transduction was enhanced by ...
Thalassaemia is a severe genetic disease that is characterised by significantly reduced production of functional beta-globin, a component of haemoglobin, the oxygen-carrying protein in the blood.