In fact, we have long known how to live a healthy life: a balanced diet, regular exercise and less stress help to avoid heart disease. And they are known to be the number one cause of death in the world. But what if you could also get vaccinated against the most important coronary diseases? And with a vaccine that changes its own DNA in such a way that lifelong protection is guaranteed?
Researchers claim that this vision may not be far off. Major advances in gene editing, particularly in CRISPR technology, could make this possible in the foreseeable future. In the early days, CRISPR was used to cut away sections of DNA, hence the colloquial term “gene scissors”. Today it is also being tested as a way to alter existing genetic code and even insert entirely new stretches of DNA, or possibly entire genes, into the human genome.
The new techniques mean that CRISPR could potentially help treat many more diseases — including some that aren’t all genetic. For example, in July 2022, Verve Therapeutics launched a study of a CRISPR-based therapy designed to alter a person’s genetic code to permanently reduce cholesterol levels. The first recipient of the therapy – a volunteer in New Zealand – had a hereditary risk of high cholesterol and already had heart disease. However, Kiran Musunuru, co-founder and chief scientific advisor at Verve, believes his company’s approach could help almost anyone.
The evolution of CRISPR
While other innovations are still being researched in petri dishes and laboratory animals, CRISPR treatments have already been tested on humans. This is astonishing if only because the technology was first used around ten years ago to modify a cell genome. “It was a pretty quick route to the clinic,” says Alexis Komor of the University of California, San Diego, who pioneered some of the newer forms of CRISPR gene editing.
Such treatments usually work by directly altering the DNA in the genome. The first generation of CRISPR technology essentially involves making cuts in the DNA. The cells themselves then repair these interventions – and the process usually prevents an existing harmful gene mutation from having negative effects. Newer forms of CRISPR work in a slightly different way. For example, there is the so-called base editing, which researchers also refer to as “CRISPR 2.0”. This technique targets the core building blocks of DNA, its bases.
There are four bases in DNA, labeled A, T, C, and G. Instead of cutting up the DNA, CRISPR 2.0 machines can convert one of those letters into another. Base editing can swap a C for a T or an A for a G. “It no longer works like scissors, but more like a pencil with an eraser,” explains Musunuru. In theory, base editing should be even safer than the original form of CRISPR gene editing. Because the DNA isn’t cut, there’s less chance of accidentally turning off an important gene or of the DNA being incorrectly reassembled by the cells’ clean-up activities.
