ArticleBlood2026
Therapeutic base editing to generate a gain-of-function F9 variant for hemophilia B.
Article in Blood, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
3 citing papers in PubMed.
- Engineering a compact high-fidelity Staphylococcus aureus Cas9 variant with broader targeting range and mechanistic insights into its activation.Nature communications · 2026Article
- Advancements in CRISPR-basedFrontiers in genome editing · 2026Review
- "An integrated multitool analysis contributes elements to interpreting unclassified factor IX missense variants associated with hemophilia B": comment from Lee et al.Journal of thrombosis and haemostasis : JTH · 2025Article
Corrections and comments
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Authors and funding
17 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
abstractThe repair of pathological gene variants is an ultimate goal in treating genetic diseases; however, developing distinct therapeutic reagents for each of the numerous variants within a gene may not be scalable. Here, we investigated whether base editing to introduce a gain-of-function variant in blood coagulation factor IX (FIX) can increase FIX activity as a targeted therapeutic approach for hemophilia B. We engineered a G:C to A:T substitution at c.1151 of F9 by cytosine base editing to generate R338Q (the Shanghai F9 variant), which markedly increases coagulation factor activity. An adeno-associated virus vector harboring the base editor converted >60% of the target G:C to A:T and increased FIX activity in HEK293 cells harboring patient-derived F9 variants as well as in knock-in mice carrying a human F9 complementary DNA. Furthermore, administration of lipid nanoparticles containing the base-editor mRNA and guide RNA increased FIX activity in mice. These data indicate that cytosine base editing to generate R338Q in FIX is a broadly applicable genome-editing strategy for hemophilia B with residual FIX activity.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.