ArticleScience advances2022
In vivo delivery of CRISPR-Cas9 using lipid nanoparticles enables antithrombin gene editing for sustainable hemophilia A and B therapy.
Article in Science advances, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 101 papers.
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101 citing papers in PubMed, 174 citations in OpenAlex.
- Optimizing twin prime editing components for scalable genome editing and therapy in spinocerebellar ataxia type 3.Molecular therapy. Nucleic acids · 2026Article
- Preparation of targeted lipid nanoparticles for precision nucleic acid delivery.Nature protocols · 2026Review
- Mutation-agnostic base editing of the progerin farnesylation site rescues Hutchinson-Gilford progeria syndrome phenotypes in neuromuscular organoids.Nature communications · 2026Article
- Dual-mode application of transcriptional regulation and DNA editing using the ultra-compact TnpB system.Nucleic acids research · 2026Article
- RNA delivery to the corneal endothelium using charge-altering releasable transporters.Science advances · 2026Article
- Identification of a cell subpopulation with different response to lipid nanoparticles and effect of protein corona on uptake and transfection.Materials today. Bio · 2026Article
- Resolving Heterogeneity of Targeted Lipid Nanoparticles Through Solution-Based Biophysical Analyses.Advanced materials (Deerfield Beach, Fla.) · 2026Article
- [Exploring the feasibility and limitations of using large language model to interpret bibliometric findings: a case study in hemophilia gene therapy].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2026Article
- Nanoengineering Systems for Gene Therapy: Mechanisms, Modalities, and Future Directions.International journal of molecular sciences · 2026Review
- Enhanced Intracellular Stability and Translation Efficiency of mRNA Drugs by a 2-arm mRNA Platform.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- sgRNA amount is a limiting factor in adenine base editing using RNA LNPs.Molecular therapy. Nucleic acids · 2026Article
- Review
- Resolving heterogeneity of targeted lipid nanoparticles through solution-based biophysical analyses.bioRxiv : the preprint server for biology · 2026Article
- CRISPR-Cas9 Therapeutics in Early Clinical Development: Delivery and Molecular Diagnostics.Cells · 2026Review
- Dual SORT LNPs for multi-organ base editing.Nature biotechnology · 2026Article
- In vivo genome editing of human haematopoietic stem cells for treatment of blood disorders using mRNA delivery.Nature biomedical engineering · 2026Article
- The mRNA-Based Innovative Strategy: Progress and Challenges.Nano-micro letters · 2026Review
- Establishment of CRISPR-Cas9-Mediated Gene Editing in the Swimming CrabMolecules (Basel, Switzerland) · 2026Article
- Advancements in CRISPR-basedFrontiers in genome editing · 2026Review
41 more citing papers are in PubMed but not listed here.
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Authors and funding
19 authors at 4 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemophilia is a hereditary disease that remains incurable. Although innovative treatments such as gene therapy or bispecific antibody therapy have been introduced, substantial unmet needs still exist with respect to achieving long-lasting therapeutic effects and treatment options for inhibitor patients. Antithrombin (AT), an endogenous negative regulator of thrombin generation, is a potent genome editing target for sustainable treatment of patients with hemophilia A and B. In this study, we developed and optimized lipid nanoparticles (LNPs) to deliver Cas9 mRNA along with single guide RNA that targeted AT in the mouse liver. The LNP-mediated CRISPR-Cas9 delivery resulted in the inhibition of AT that led to improvement in thrombin generation. Bleeding-associated phenotypes were recovered in both hemophilia A and B mice. No active off-targets, liver-induced toxicity, and substantial anti-Cas9 immune responses were detected, indicating that the LNP-mediated CRISPR-Cas9 delivery was a safe and efficient approach for hemophilia therapy.
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