Gene editing saves two children

摘要

Two girls given gene-edited cells to kill their cancers are both doing well more than a year later. An 11-month-old girl called Layla was the first to get the treatment, in June 2015, followed by a 16-month-old girl who was given cells in December 2015. The treatment is a form of CAR T-cell therapy. This involves using a virus to add a gene to immune cells to make them target specific cancers. The approach normally requires people to be treated with their own modified immune cells. If other cells are used, they can attack the patient's body. However, it can be difficult to extract enough immune cells from very young or ill patients.