A Simple Way To Turn Genes On Or Off May Change Medicine


Depending on who you are, CRISPR Cas9, a tool for editing genes recently approved to be used on humans, will either usher in a new age of medicine or mark the end of humanity as we know it as we modify ourselves into horrific new forms. And it’s unlikely that genetic editing, whether for good or bad, is a debate we’re going to end any time soon. But what if we could treat genetic disease without genetic editing? Scientists at the Jonas Salk Institute are doing just that.

The Salk Institute used CRISPR Cas9 not to remove genes, but to turn them on or shut them off, using epigenetics. Think of it this way: The genome is an instrument, and genetic editing is like tuning that instrument. Epigenetics are the song being played on that instrument, with genetic diseases being the sour notes. You’re not changing the instrument at all, you’re just getting some help playing it better.

That’s especially important because epigenetic drugs have been notoriously imprecise. To continue the metaphor, they try to play a note and more often than not slam their whole hand on the keyboard. But while CRISPR Cas9 is not quite perfect in that regard, it’s much, much more precise than other epigenetic tools, and that accuracy could make a world of difference. Being able to silence, or activate, certain genes would help mitigate the misery these diseases can cause and in some cases wipe them out completely, all without changing the patient’s genome and with a much lower chance of side effects. Also helping matters is, well, nobody’s ever made a movie about epigenetics. It’s a lot easier to sell somebody on a medicine when it doesn’t instantly make them think of Gattaca.

This won’t be in use tomorrow. It’s only been tested, so far, on mice, and the human trials will necessarily be more rigorous and careful than the already rigorous mouse trials. But everything involved has already been through the FDA process once, and, barring any unforeseen problems that may emerge, we may be on the cusp of a new moment in medicine.

(via Gizmodo)