Our quest to understand how our genes work started in earnest in the crispr companies to invest in-19th Century when a biologist and monk called Gregor Mendel came to a startling conclusion about the traits of plants. He crossed purple flowered pea plants with white ones, and found that all the resulting offspring were purple. However, he noticed that the third generation produced both colours.
This revealed that a characteristic such as colour can be inherited, with one trait more dominant than another. In a way, Mendel had figured out what genes did, but not what they were, or even looked like. It was only in the following century that the very structure of DNA was discovered. Building on the work of Rosalind Franklin and Maurice Wilkins, in 1953 James Watson and Francis Crick discovered that our DNA is formed in a double helix.
Knowing this structure helped unlock more secrets. When DNA is replicated, this helix splits in two – it “unzips”. This means mutations can be introduced as our cells divide. Even a small genetic error can cause a devastating disease. In other words, the unique book of letters that makes up each of us can be printed or rewritten with mistakes. But we now have the tools – including the ability to analyse large data sets – to both read our book quicker, cheaper and even tinker with it. Let’s start with the people who are interested in doing that tinkering.
Scientists are now able to edit genes from organisms. It is now used in labs all over the world, altering and manipulating the genes of plants and animals with the idea that it can soon be used to treat numerous human diseases. The question that the public are very interested in is the possibility of using CRISPR gene editing for therapeutic purposes,” says Prof Robin Ali of the European Society for Gene and Cell Therapy. This could happen within the next decade if early studies show promise. Still, scientists remain excited about the technology as it could provide effective therapies for conditions that are currently untreatable, such as Huntington’s disease and cystic fibrosis, to name but two.
In theory CRISPR could provide treatments quickly, taking days or weeks rather than months. There are very few examples where new technology has swept across labs around the world, where it’s implemented to do things that were extremely difficult to do,” says Prof Ali. The use of CRISPR will not, however, be “instant” he warns. It will take a number of years for the technology to be used clinically. Intellia Therapeutics is one of several companies developing the technology for use in humans.
The company’s CEO, Nessan Bermingham, believes that CRISPR has the potential to completely revolutionise healthcare. The eventual hope is that it could target both diseases caused by only one defective gene as well as diseases caused by more than one genetic mutation. This technology has the potential to allow us to target multiple regions of DNA at the same time,” says Bermingham. This is unprecedented in our industry. Nessan Bermingham, Intellia Before it can be used in humans, any drugs will have to be extensively trialled and regulated by the relevant authorities.
Until then its use will mainly be as a research tool in the lab. Without doubt, the power is how easy it is to edit genomes,” says Prof Ali. Many scientific questions still have to be answered before Intellia can seek approval for clinical trials on humans. For this reason Bermingham is hesitant to propose a specific timeline.