Article

Universal base editing for hemophilia B

2024-11-15

Abstract excerpt

The repair of pathological gene variants is an ultimate aim for treating genetic diseases; however, it is not practical to develop different therapeutic reagents for each of the many variants that can occur in a gene. Here, we investigated whether base editing to induce a gain-of-function variant in blood coagulation factor IX (FIX) can increase FIX activity as a treatment strategy for hemophilia B. We engineered...

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Identifiers and source

Literature Corpus work: 8daa1a34-49f1-51cd-9875-9d771d59db96
DOI: 10.1101/2024.11.13.623331

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