ArticleNature communications2022
Low-dose AAV-CRISPR-mediated liver-specific knock-in restored hemostasis in neonatal hemophilia B mice with subtle antibody response.
Article in Nature communications, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed, 35 citations in OpenAlex.
- Functional editing of the OTC locus by targeted integration with phenotype correction and restoration of endogenous expression patterns.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
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- Advancements in CRISPR-basedFrontiers in genome editing · 2026Review
- AAV-mediated gene therapy for the CNS: The case for early treatment.Molecular therapy. Methods & clinical development · 2025Article
- Peeling back the layers of immunogenicity in Cas9-based genomic medicine.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Genomic insertion of ancestral uricase into human liver cells to determine metabolic consequences of pseudogenization.Scientific reports · 2025Article
- Trophectoderm-specific gene manipulation using adeno-associated viral vectors.Experimental animals · 2025Article
- Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Advancements in CRISPR/Cas systems for disease treatment.Acta pharmaceutica Sinica. B · 2025Review
- The evolving landscape of NF gene therapy: Hurdles and opportunities.Molecular therapy. Nucleic acids · 2025Review
- Enhancing hemophilia A gene therapy by strategic F8 deletions in AAV vectors.Blood science (Baltimore, Md.) · 2025Article
- CRISPR and gene editing technologies for bleeding disorders.Therapeutic advances in hematology · 2025Review
- Lipid Nanoparticles Enable Efficient In Vivo DNA Knock-In via HITI-Mediated Genome Editing.Biomolecules · 2024Article
- Tropism of adeno-associated virus serotypes in mouse lungs via intratracheal instillation.Virology journal · 2024Article
- Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.Cell reports. Medicine · 2024Article
- Targeted Gene Insertion: The Cutting Edge of CRISPR Drug Development with Hemophilia as a Highlight.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024Review
- Review
- Iron oxide-coupled CRISPR-nCas9-based genome editing assessment in mucopolysaccharidosis IVA mice.Molecular therapy. Methods & clinical development · 2023Article
- Article
- Innovations in RNA therapy for hemophilia.Blood · 2023Review
Corrections and comments
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Authors and funding
17 authors at 4 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
AAV-delivered CRISPR/Cas9 (AAV-CRISPR) has shown promising potentials in preclinical models to efficiently insert therapeutic gene sequences in somatic tissues. However, the AAV input doses required were prohibitively high and posed serious risk of toxicity. Here, we performed AAV-CRISPR mediated homology-independent knock-in at a new target site in mAlb 3'UTR and demonstrated that single dose of AAVs enabled long-term integration and expression of hF9 transgene in both adult and neonatal hemophilia B mice (mF9 -/-), yielding high levels of circulating human Factor IX (hFIX) and stable hemostasis restoration during entire 48-week observation period. Furthermore, we achieved hemostasis correction with a significantly lower AAV dose (2 × 10
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Registered trials
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