Next month, Chinese researchers will edit adult human DNA using the revolutionary CRISPR/Cas-9 tool, commonly known as CRISPR, for the first time anywhere in the world.
The researchers will attempt to cut faulty DNA out of the cells of lung cancer patients who have failed to respond to all other conventional treatments.
Chinese scientists have previously used CRISPR on non-viable human embryos, without much luck, but this is the first time any researchers, anywhere in the world, will use the tool to edit DNA in an adult.
If successful, the hope is that it could lead to further CRISPR treatments – CRISPR/Cas-9 allows researchers to effectively cut and paste DNA in cells, and, in animals, it’s already been shown to treat genetic diseases such as Duchenne muscular dystrophy.
The new trial will begin at Sichuan University’s West China Hospital next month, according to Nature, and will involve patients who’ve already gone through chemotherapy and radiation therapy – in other words, are out of options.
There are also concerns that it could lead to the development of ‘designer’ babies – where parents pick and choose certain traits to write into their child’s DNA.
But it’s important to note that the new Chinese study will only edit patients’ immune system T-cells, and not affect gamete cells that could be passed down to offspring.
In other words, the changes made by CRISPR will be limited to the patient involved in the trial – a number that hasn’t been disclosed as yet.
We do know what the process will involve, though.
In the trial, the Chinese scientists will extract T-cells from patients’ blood and delete a gene that produces a protein called PD-1, which stops T-cells from targeting and killing cancer cells, from their DNA.
The team will then multiply these new CRISPR-modified T-cells in the lab, before injecting them back into the patients to flood their immune system.
The hypothesis is that, with PD-1 inhibited, the T-cells will be able to track down and wipe out lung cancer cells naturally.
While CRISPR/Cas-9 is capable of also inserting new DNA into a cell’s genome, in this study, genetic information will only be deleted, not added.
This is a similar process to immunotherapy studies that are already common around the world – researchers take immune cells, genetically modify them, and insert them back into patients. In fact, gene editing has already saved the life of a girl with ‘incurable’ leukaemia.
But what’s different in this case is the use of CRISPR, which is incredibly simple and versatile.
While in the past it’s taken years for scientists to develop the right molecular ‘scissors’ to cut out specific genes, CRISPR simply needs to be programmed and can then work for any part of the genome – no costly development required.
Last month, the US National Institutes of Health (NIH) approved a similar trial in the states – although the American research will also add an extra gene to help combat three types of cancer: melanoma, sarcoma, and myeloma.
The research could begin as early as this year, but if reports are anything to go off, China will be the first to try out this incredibly powerful tool in humans.