A team led by Professor Paolo De Coppi has announced that they are set to build and transplant an oesophagususing organs harvested from pigs and then modified using a child’s stem cells.The organs will be created for children who are born with a severe cases of oesophageal atresia, where their oesophagus has not formed properly.
The UCL Technology Fund (UCLTF) has approved an investment of up to £1 million to support researchers Professor Adrian Thrasher, Professor Bobby Gaspar, and Dr GiorgiaSantilli in developing a gene therapy approach for treating p47phox-deficient chronic granulomatous disease (p47-CGD).
In a European first, a little-known gland called the ‘thymus’, some of which is routinely removed during cardiac surgery, has saved the lives of children with a life threatening immunodeficiency condition, complete DiGeorge syndrome (cDGS).
Great Ormond Street Hospital (GOSH) has been announced as the lead in a new Europe-wide epilepsy network, aiming to increase collaborations across Europe and improve access to innovative and highly-specialised diagnostics.
At the end of 2016, the American Food and Drug Administration (FDA) granted approval for the use of Spinraza (nusinsersen) for the treatment of spinal muscular atrophy (SMA) in paediatric and adult patients. This is unprecedented for the SMA community witnessing the first approved drug for this disease.
Research carried out by BRC-funded Professor Paul Brogan and his team has defined a new autoinflammatory disease (AID) in humans with periodic fevers, immunodeficiency and intermittent thrombocytopenia.