[roncesvalles]

CRIPSR was a game-changer for genetics research. A lot of gene knockout studies use CRISPR. However, it was always weirdly overhyped for clinical use from the beginning and this was obvious to anyone with a genetics background.

The public in general doesn't have a good understanding of basic genetics and I blame high school science curriculums for not covering it well enough. Too much time is wasted on Mendelian genetics without covering the Central Dogma.

You basically cannot "edit" your somatic DNA in a meaningful wholesale way since every single cell in your body has a copy of the DNA, and it's a foolish endeavor. What you can conceivably edit to good effect is your germline DNA, stem cell DNA, or modify mRNA expression (e.g. retinoids; yes putting retinol/adapalene cream on your face is "gene therapy"), or introduce foreign mRNA for your translation machinery to co-opt (e.g. mRNA vaccines).

[Bjartr]

Edit every cell? No. Edit enough cells to impact health outcomes for a meaningful period of time? [Yes](https://www.youtube.com/watch?v=J3FcbFqSoQY)

[shevy-java]

This approach can work for some genetic diseases such as blindness based on some cells in the retina or partial blindness. For others this is not really a cure. If you want to cure people with progeria, does curing 20% of the cells really help? Perhaps 100% is not necessary, but it would seem strange to cure only some cells but not others. You'd have a mosaic of cells where some would work and others don't. Cells interact; timing also plays a role in development. I don't really see that aiming for anything but a very high number of cells cured, can work.

[projektfu]

I disagree that it's "gene therapy" to affect the natural regulation of mRNA production. If that were true then the term "gene therapy" loses its meaning, as just about everything changes the expression of mRNA. You can probably do so somewhere just by thinking really hard about it.

Expressing mRNA that doesn't exist in the genome, that would be gene therapy. Or just a virus.

[asdff]

It was a game changer in terms of making things cheaper and a little easier. However the actual functionality was still possible with other methods. Zinc finger nucleases for example. Knockdown via RNAi is often still done because a knockout target may be inviable, and it is pretty cheap and easy to knockdown in most model systems.