Critically, the gene editing of the red blood cells can be done in the lab; trying to devise an injectable or oral substance that would actually transport the gene-editing machinery to an arbitrary part of the body is much harder.
I am totally confused by this. Mature red blood cells don’t contain a nucleus, and hence no DNA. There is nothing to edit. Injecting blood cells produced by gene-edited bone marrow in vitro might work, but would only be a therapy, not a cure: it would have to be repeated regularly. The cure would be to replace the bone marrow.
So I resorted to reading through the linked FDA article. Relevant section:
The modified blood stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow and increase the production of fetal hemoglobin (HbF), a type of hemoglobin that facilitates oxygen delivery.
Blood stem cells seems to be FDA jargon for ematopoietic stem cells. From the context, I would guess they are harvested from the bone marrow of the patients, then CRISPRed, and then injected back in the blood stream where they will find back to the bone marrow.
I still don’t understand how they would outcompete the non-GMO bone marrow which produces the faulty red blood cells, though.
I would also take the opportunity to point out that the list of FDA-approved gene therapies tells us a lot about the FDA and very little about the state of the art. This is the agency which banned life-saving baby nutrition for two years, after all. Anchoring what is technologically possible to what the FDA approves would be like anchoring what is possible in mobile phone tech to what is accepted by the Amish.
Also, I think that editing of multi-cellular organisms is not required for designer babies at all.
Start with a fertilized egg, which is just a single cell. Wait for the cell to split. After it has split, separate the cells into two cells. Repeat until you have a number of single-cell candidates.
Apply CRISPR to these cells individually. allow them to split again. Do a genome analysis on one of the two daughter cell. Select a cell line where you did only the desired change in the genome. Go back to step one and apply the next edit.
Crucially, the costs would only scale linearly with the number of edits. I am unsure how easy is that “turn one two-cell embryo into two one-cell embryos”, though.
Of course, it would be neater to synthesize the DNA of the baby from the scratch, but while prices per base pair synthesis have been dropped a lot, they are clearly still to high to pay for building a baby (and there are likely other tech limitations).
I am totally confused by this. Mature red blood cells don’t contain a nucleus, and hence no DNA. There is nothing to edit. Injecting blood cells produced by gene-edited bone marrow in vitro might work, but would only be a therapy, not a cure: it would have to be repeated regularly. The cure would be to replace the bone marrow.
So I resorted to reading through the linked FDA article. Relevant section:
Blood stem cells seems to be FDA jargon for ematopoietic stem cells. From the context, I would guess they are harvested from the bone marrow of the patients, then CRISPRed, and then injected back in the blood stream where they will find back to the bone marrow.
I still don’t understand how they would outcompete the non-GMO bone marrow which produces the faulty red blood cells, though.
I would also take the opportunity to point out that the list of FDA-approved gene therapies tells us a lot about the FDA and very little about the state of the art. This is the agency which banned life-saving baby nutrition for two years, after all. Anchoring what is technologically possible to what the FDA approves would be like anchoring what is possible in mobile phone tech to what is accepted by the Amish.
Also, I think that editing of multi-cellular organisms is not required for designer babies at all.
Start with a fertilized egg, which is just a single cell. Wait for the cell to split. After it has split, separate the cells into two cells. Repeat until you have a number of single-cell candidates.
Apply CRISPR to these cells individually. allow them to split again. Do a genome analysis on one of the two daughter cell. Select a cell line where you did only the desired change in the genome. Go back to step one and apply the next edit.
Crucially, the costs would only scale linearly with the number of edits. I am unsure how easy is that “turn one two-cell embryo into two one-cell embryos”, though.
Of course, it would be neater to synthesize the DNA of the baby from the scratch, but while prices per base pair synthesis have been dropped a lot, they are clearly still to high to pay for building a baby (and there are likely other tech limitations).