Single prime editing system could potentially treat multiple genetic diseases

I found this new article from the Broad Institute super interesting because it talks about a way to fix many different genetic diseases using one editing system by a pioneer, David Liu. Usually, gene therapy focuses on fixing each individual mutation, which is why it takes forever for treatments to get developed. But the scientists at the Broad might have found a way around that.

They designed something called PERT, which stands for Prime Editing–Mediated Readthrough of Premature Termination Codons. Basically, instead of correcting every single DNA mutation, PERT targets a whole type of mutation called a nonsense mutation—which is when a DNA change makes a stop codon appear too early. That stop codon shuts down protein production and causes a bunch of different diseases. Since nonsense mutations cause almost 30% of all known genetic disorders, being able to treat them together is a huge deal.

What PERT does is insert a special suppressor tRNA using prime editing. This tRNA tells the ribosome to ignore the bad stop codon so the cell can actually finish making the full protein. What’s cool is that once the suppressor tRNA is added, the cell can potentially fix many nonsense mutations, not just one.

The research team tested it in human cells from several rare diseases (like Tay-Sachs, Batten disease, and Niemann-Pick). They got 20–70% of normal protein function back, which is honestly pretty impressive. They also tested it in mice with Hurler syndrome, and even though it only restored about 6% activity, it still improved the disease symptoms a lot.