[crakenzak]

Yup Casgevy & Lyfgenia! These sickle cell (and now also approved for beta thalassemia) CRISPR therapies basically work in the following way:

- Blood stem cells are removed from the patient and the CRISPR Cas9 protein outside of the body is injected to cut the gene responsible for suppressing fetal hemoglobin production (even people with sickle cell have healthy fetal hemoglobin, their adult hemoglobin gene is what causes the deformed red blood cells)

- Chemotherapy is used to kill all living bone marrow and remove all previous unedited stem cells.

- New edited stem cells are inserted, and patient recovers with new blood production being of healthy red blood cells.

I'd say a huge step forward was FDA and EMA approval, but figuring out a way to remove previous unedited stem cells with chemotherapy would be a step change in the patient experience.

[Etheryte]

It's both extremely sci-fi and incredibly terrifying that one of the steps for a cure is to quote unquote kill all living bone marrow. Modern medicine is fascinating in how advanced it can be.

[l33tman]

Yeah it's a spectacular transplant procedure, though it's really not that modern medicine, it has been developed since the 50s! The step where you irradiate or apply chemo to kill off the existing bone marrow had to be done at first because the patients had leukemia so you had to do this anyway. An interesting thing is that if there are still cells left in you, they will be wiped clean by the new transplanted immune system in a so called graft-vs-host response that also sounds like a horror-movie concept :)

[blkhawk]

not sure if you need to kill absolutely all living bone marrow. Unlike cancer having 5% sickle cell red blood cells is probably fine. That makes the chemo probably way less bad than what you would need for cancer.

[renegade-otter]

And yet, measles are back.

[giantg2]

"- Chemotherapy is used to kill all living bone marrow and remove all previous unedited stem cells.

- New edited stem cells are inserted, and patient recovers with new blood production being of healthy red blood cells."

It's very likely HIV could be cured similarly. I believe all the people who have been cured so far are bone marrow transplant recipients in which the marrow had a specific gene or genes.