How does CRISPR gene editing work to treat diseases?

CRISPR gene editing is like having a special tool that lets doctors fix broken parts of our body at the very tiny level of genes, which are like instruction books for how our cells work.

Imagine your body is like a big machine, and each part of it has its own set of instructions written on paper. If one of those papers gets torn or has a typo, that part of the machine might not work right, just like how you might have trouble reading if your book had smudged letters.

That’s what happens in some diseases, the instruction books (genes) get messed up. With CRISPR, doctors can find the mistake and fix it, like using a pair of scissors to cut out the wrong part and replace it with the right one.

CRISPR uses a molecular scissors, called Cas9, which are guided by a special kind of map, like a GPS for the cell. This map tells Cas9 exactly where to go in the instruction book so it can cut out the bad part.

Once the mistake is removed, scientists can add the correct instructions back in, or let the cell fix itself. That way, the body starts working better again, just like when you fix a torn page in your favorite storybook and it becomes readable once more!