3 ms·

There's a sickle cell therapy that uses CRISPR. I don't know the specifics but if one has been successful then a second can be too. I'm looking forward to gett

There's a sickle cell therapy that uses CRISPR. I don't know the specifics but if one has been successful then a second can be too. I'm looking forward to getting more information in the future. Cell culture vs cure is a long road but at least there's hope. The big drawback is that these therapies are super expensive but time and experience in the past have proven that the costs can be lowered. "Sickle Cell Disease Approvals Include First CRISPR Gene Editing Therapy" https://jamanetwork.com/journals/jama/article-abstract/2813761#:~:text=Casgevy%E2%80%94the%20CRISPR%2DCas9%20gene,preventing%20painful%20vaso%2Docclusive%20crises https://jamanetwork.com/journals/jama/article-abstract/28137....

crakenzak 3y ago
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 3y ago
  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 3y ago
    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 :)
  - renegade-otter 3y ago
    And yet, measles are back.
  - blkhawk 3y ago
    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.
- giantg2 3y ago
  "- 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.
saati 3y ago
It's very different, you only have to fix some of the bone marrow to help someone with sickle-cell, but you have to eliminate all reservoirs of HIV to cure AIDS.
- WheelsAtLarge 3y ago
  I was under the understanding that HIV attacks only immune cells, white blood cell, only. All the issues that eventually kill are due to the body not being able to defend itself. So it seems like a cure would be very much similar to a sickle cell cure. Is that not the case?
dghughes 3y ago
A good video explanation of that too via Sci Show. It's quite complex; chemo, lab work and takes a long time and not always successful but the majority of the time it is successful. https://www.youtube.com/watch?v=uHWD8RSw4As https://www.youtube.com/watch?v=uHWD8RSw4As

crakenzak 3y ago

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 3y ago
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 3y ago
  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 :)
- renegade-otter 3y ago
  And yet, measles are back.
- blkhawk 3y ago
  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.
giantg2 3y ago
"- 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.

saati 3y ago

It's very different, you only have to fix some of the bone marrow to help someone with sickle-cell, but you have to eliminate all reservoirs of HIV to cure AIDS.

WheelsAtLarge 3y ago
I was under the understanding that HIV attacks only immune cells, white blood cell, only. All the issues that eventually kill are due to the body not being able to defend itself. So it seems like a cure would be very much similar to a sickle cell cure. Is that not the case?

dghughes 3y ago

A good video explanation of that too via Sci Show. It's quite complex; chemo, lab work and takes a long time and not always successful but the majority of the time it is successful. https://www.youtube.com/watch?v=uHWD8RSw4As https://www.youtube.com/watch?v=uHWD8RSw4As