Next step for CRISPR: gene editing for the masses?
That vision is not far off, say the researchers. Advances in gene editing, and CRISPR technology in particular, may soon make this possible. In the beginning, CRISPR was used to simply make cuts in DNA. Today, it’s being tested as a way to alter the existing genetic code, even inserting whole new pieces of DNA or possibly entire genes into someone’s genome.
These new techniques mean that CRISPR could potentially help treat many other conditions, not all of which are genetic. In July 2022, for example, Verve Therapeutics launched a trial of a CRISPR-based therapy that alters the genetic code to permanently lower cholesterol levels.
The first beneficiary, a volunteer in New Zealand, has a hereditary risk of high cholesterol and already suffers from heart disease. But Kiran Musunuru, co-founder and chief scientific adviser at Verve, thinks the approach could help almost anyone.
The treatment works by permanently turning off a gene that codes for a protein called PCSK9, which appears to play a role in maintaining blood cholesterol levels.
“Even if you start with normal cholesterol and turn off PCSK9 and lower cholesterol even further, it reduces the risk of having a heart attack,” says Musunuru. “It’s a general strategy that would work for anyone in the population.”
The evolution of CRISPR
While new innovations are still being explored in lab dishes and research animals, CRISPR treatments have already entered human trials. This is a staggering achievement considering that the technology was first used to edit cell genomes around 10 years ago. “It was a pretty quick trip to the clinic,” says Alexis Komor of the University of California, San Diego, who developed some of these new forms of CRISPR gene editing.
Gene editing treatments work by directly modifying the DNA of a genome. The first generation of CRISPR technology essentially cuts DNA. Cells repair these cuts, and this process usually prevents a harmful genetic mutation from having an effect.
Newer forms of CRISPR work slightly differently. Take the basic edition, which some describe as “CRISPR 2.0”. This technique targets the basic building blocks of DNA, called bases.