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Transformational therapy cures haemophilia B

4 pointsby distant_hatalmost 3 years ago

2 comments

ggmalmost 3 years 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?
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distant_hatalmost 3 years ago
The actual paper. Unfortunately paywalled.<p>[1] <a href="https:&#x2F;&#x2F;www.nejm.org&#x2F;doi&#x2F;full&#x2F;10.1056&#x2F;NEJMoa2119913?query=recirc_top_ribbon_article_4" rel="nofollow">https:&#x2F;&#x2F;www.nejm.org&#x2F;doi&#x2F;full&#x2F;10.1056&#x2F;NEJMoa2119913?query=re...</a>