An international collaboration has shown the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells have been cleared by the body.
Rare Disease Day is an international day celebrated in over 100 countries to raise awareness amongst the general public, the clinical community, and decision-makers about rare diseases and their impact on people affected and their families.
Ahead of Rare Disease Day 2021, GOSH will join forces with three other leading children’s research institutions on three continents to decipher paediatric illnesses, including rare diseases, and find better treatments.
Great Ormond Street Hospital for Children NHS Foundation Trust (GOSH) is playinga key role in the world’s first COVID-19 human challenge study, which will get underway shortly following approval from the UK’s clinical trials ethics body.
Scientists at the UCL GOS Institute of Child Health reveal that new genetic mutations which occur during embryonic development can cause the severe birth defect spina bifida. Many genes have been implicated in spina bifida, and mutations which are n…