Scientists have used a genome-editing technique to cure mice of an inherited liver disease, raising the prospect that similar clinical trials on humans could begin within a few years.
Britain’s Independent reports that the scientists at MIT used a technology called Crispr, which allows them to alter DNA at specific points of the chromosomes of animals or plants. The researchers altered a single “letter” of the genetic alphabet that had been mutated in a gene involved in liver metabolism.
The paper reports that the equivalent liver disease is caused in humans by a similar mutation in the same gene. The word “letters” in this case refers to the 3 billion “base pairs” of the DNA molecule. Correcting a single base pair, as the MIT scientists did to cure the mice, requires a level of exactitude equivalent to correcting a single word in a dictionary.