dv said:
http://www.sciencealert.com/gene-editing-has-saved-the-life-of-a-girl-with-leukaemia
Gene editing has saved the life of a girl with “incurable” leukaemia
“Researchers in the UK have successfully treated Layla, a baby girl with leukaemia, using a gene-editing therapy that had previously only been trialled in mice. Doctors say it’s too soon to declare her cured, but she’s now living healthily and cancer free.
The therapy involved using mass-produced gene-edited cells to attack the leukaemia in her body, and it was the first time it had been tried in a human. But because Layla’s case had been declared incurable and doctors had exhausted all other options, her parents were granted permission to use the experimental treatment on compassionate grounds”
> Normally gene editing works by taking immune T-cells from a patient’s body and then genetically engineering them to attack cancerous cells before placing them back in the body.
That works?!
> This is already being trialled in humans, and results so far suggest that it can be effective. But it won’t work for patients like Layla, who have already undergone extensive treatment for leukaemia and don’t have enough healthy T-cells left to work with.
> A healthy person donates a whole bunch of T-cells, which are then modified to make sure they’re safe to transfer, creating what the researchers call UCART19 cells. To create these cells, Qasim and his team use a pair of ‘molecular’ scissors, known as TALEN proteins, to switch off certain receptors, ensuring that the UCART19 T-cells only attack leukaemia cells, and not healthy ones.
Amazing.
> They also removed genes to make the cells invisible, so that they wouldn’t be destroyed by other leukaemia drugs. “The approach was looking incredibly successful in laboratory studies,” said Qasim, “and so when I heard there were no options left for treating this child’s disease, I thought ‘why don’t we use the new UCART19 cells?’”
Superb. Is there any limit to what this can do, with leukemia?
Other web reports on UCART19 and TALEN.
Technical extended abstract, same case
Press release about same case
A huge advantage of this is that it can be done “off-the-shelf”, a single product that could help hundreds if not thousands of leukemia sufferers that doesn’t have to be tailor-made for each patient.
Wikipedia on TALENs
I went looking for a paper on the testing of this therapy on animal studies and didn’t find any. Looks like they went straight from cell culture to human without testing on animals first. Good on them, apart from the ethical implications, animal testing could have slowed down implementation for up to a decade.