A 13-year-old girl whose leukaemia had not responded to other treatments now has no detectable cancer cells after receiving a dose of immune cells that were genetically edited to attack the cancer
This approach is now approved in the UK for people with leukaemia that involves so-called B cells, another type of immune cell. Alyssa’s leukaemia was caused by T cells and if CAR-T cells are modified to attack other T cells, they just kill each other.
Qasim’s team therefore made an additional change to the CAR-T cells by knocking out the gene for the receptor that identifies them as T cells. Creating these CAR-T cells requires making four gene edits at once, which leads to yet another problem. Conventional gene editing involves cutting DNA strands and relying on a cell’s repair machinery to rejoin the ends. When lots of cuts are made at once, cells sometimes die. Even if they survive, the wrong ends can be put back together, leading to major mutations that can potentially make the cells cancerous. The more gene edits that are made, the more likely this is to occur.
So Qasim and his team instead used a modified form of the CRISPR gene-editing protein that doesn’t cut
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