4 ms·
What's the status of just using CRISPR to fix pathogenic variants?
by strbean 4mo ago
What's the status of just using CRISPR to fix pathogenic variants?
- epistasis 4mo agoFor fully formed humans? Not great, with exceptions where you can target a 2D layer of cells that are covered in fluids. I've seen some people trying this with, say, retinas, where you only need to target some of the cells. And also with intestinal/colorectal linings, where cells reproduce quickly so there's a chance of actually replacing all of the cells eventually. But for most of the body, I don't think anybody has ever been optimistic about CRISPR fixing pathogenic variants. CRISPR is a great tool, but it doesn't help with delivering gene therapy across the body.
- strbean 4mo agoSo for masses of solid tissue, we can't effectively deliver the payload to all the cells? How about for lymphomas?