Kirthana Balachandran Kirthana Balachandran was diagnosed when she was three months old and is now studying to be a doctor The first therapy that uses gene-editing is to be offered on the NHS in a “revolutionary breakthrough” for patients. It will be used as a potential cure for the blood disorder beta thalassaemia. Stem cells … Read more
Medical regulators approve a gene therapy that aims to cure sickle cell disease and beta thalassemia. Source link
The new rules close the loopholes that enabled Dr He to evade regulations, which previously only applied to experiments on human beings in hospitals, for example for drug trials. The updated regulations cover all research institutions and everything relating to humans, including work on tissues, organs and embryonic cells. Source link
His family urged Jimi to take part in other clinical trials or have a bone marrow transplant – which is an option for some people with sickle cell. But he thought they would involve too much time in hospital for the improvement in his quality of life. Instead, he pinned his hopes on a cure … Read more