5 ms·
Transformational therapy cures haemophilia B
- distant_hat 4y agoThe actual paper. Unfortunately paywalled. [1] https://www.nejm.org/doi/full/10.1056/NEJMoa2119913?query=recirc_top_ribbon_article_4 https://www.nejm.org/doi/full/10.1056/NEJMoa2119913?query=re...
- ggm 4y ago"Transformational" as a superlative. It's gene therapy. I'd love them to explain how they make genetically modified cells implant in bone marrow, if this is like a bone marrow transplant or some encapsulated method like people are trying with insulin. Or a viral vector?
- distant_hat 4y agoThe virus vector doesn't implant in the bone marrow. Hemophilia is caused by deficiency of Factor VIII (Hemophilia A) or Factor IX (Hemophilia B). The factor is produced in the liver and they use a virus that specifically targets the liver to get the gene in there. I agree though that transformational is a superlative and there have been other gene therapies that showed in initial promise for Hemophilia but the effects fade out over a few years.
- ggm 4y agoThanks for clarifying. Given the livers reputed regenerative powers I'm surprised a change persists but it's super good for haemophilia if this works.