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comment by rooibos
rooibos  ·  4024 days ago  ·  link  ·    ·  parent  ·  post: Fyodor Urnov on Genome Editing

If the whole bone marrow is removed in an ablative treatment (rather than a partial fashion), some of the clinical risks of the treatment remain.

The big two risks with bone marrow transplants are

1) rejection of the new marrow and

2) infection, as the immune system is suppressed while the marrow regrows.

Using one's own marrow with largely one's own DNA should (?) eliminate the first to a large degree, but the second still remains.





thundara  ·  4024 days ago  ·  link  ·  

In the case of the latter, I talked to one of the researchers who worked on this project. A thing they mentioned was that, after breaking and rejoining the DNA, you don't necessarily need to screen the stem cells for those that integrated the corrected fragment. Once in a human body, at least in the cases of β-thalassemia and sickle cell anemia, there is a selective advantage for the modified stem cells.