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by akkawwakka 2198 days ago
It’s worth knowing there are very few diseases that can be cured with singular gene edits. Many diseases involve multiple genes, cascades of gene expression, or complex physiological pathways. Oh, and not to mention epigenetics and environmental factors.
3 comments

The strategy they used speaks exactly to your point. The diseases were not "cured" in the sense that these people were brought back to a generic baseline. That very well may have required a complex series of edits that are well beyond what is currently possible. Instead, they focused on a clever workaround that was simpler to implement, reactivating fetal hemoglobin. That's how they were able to cure two different diseases with the same gene edit.

So you have a much larger pool of genetic and non-genetic diseases that can be cured now. For example if someone had low lung function due to lung scarring, this might be an interesting treatment to look at.

My family has one of the well-known single-site cancer-causing mutations; we know what the specific site is, even. It would be immense to "fix" (I am not a geneticist) that single site. Even just for my kids.
Why would you prefer the risks of editing versus the risks of preimplantation screening? Editing would be strictly riskier unless there’s really no alternative (unlikely but possible)
Preimplantation screening isn't an option for me or any other of my family members who are long past implantation. I'm not saying I'd hop on stage 1 clinical trials of a potential editing cure, and we'd have to see what the data on risk looked like to compare it with the risk from cancer without editing.
Actually, there is a couple thousand monogenic Mendelian diseases, so work isn't short, also not people, just money. However, you won't treat a developmental delay with a gene edit, that only works for non-working enzymes etc. without too much permanent damage.