CTX001 is an 'ex vivo' application of gene-editing, in which the technology is used to modify a patient's own cells outside the body to make foetal haemoglobin (HbF), which can serve as a ...

CTX001 uses the CRISPR gene editing technique to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells. HbF is a form of the oxygen ...