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An uncurable disease (hemophilia type B) could be cured
| nice
| Well, that's cool.
| Finally some fucking good news
| So this uses gene therapy right?
| >>274246
kinda. Depends on what you consider a gene therapy. My understanding - they modified the genes in stem cells, put them back in the mouse to override the existing gene pattern in its liver.
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| Scientists at the Salk Institute have combined CRISPR-Cas9 gene editing with stem cell technology to generate a one-time, autologous cell therapy for the genetic blood clotting disorder hemophilia B. In vivo tests showed that gene-edited, stem cell–derived liver cells remained viable and functional in hemophiliac mice for nearly a year, after just a single injection.
>source: www.genengnews.com https://tinyurl.com/ybdj62gx