ArticleCell reports2017
CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors.
Article in Cell reports, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 85 papers, 1 of them a synthesis that pooled it.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
85 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Evidence Synthesis of Gene Therapy and Gene Editing from Different Disorders-Implications for Individuals with Rett Syndrome: A Systematic Review.International journal of molecular sciences · 2023Pooled it
- Electroporation for High-Efficiency Delivery of CRISPR to Hematopoietic Cells.Methods in molecular biology (Clifton, N.J.) · 2027Article
- Double-stranded DNA donors and CRISPR-Cas9 for universal correction of mutations causing cystic fibrosis in human airway cells.Molecular therapy. Nucleic acids · 2026Article
- AAV-mediated delivery of CRISPR/Cas9 targeting conserved overlapping ORFs efficiently suppresses HBV replication in hepatocyte models.Biotechnology reports (Amsterdam, Netherlands) · 2026Article
- Stepwise Evaluation of Plasmid- and Adeno-Associated Virus-Based Knock-In Using A Triple-Reporter Platform.Genes to cells : devoted to molecular & cellular mechanisms · 2026Article
- CAR-mediated release of IL-10 increases the function of regulatory T cells: Relevance for future clinical application.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Efficient multi-kilobase knock-ins in mice and cell lines using CRISPR/Cas9 and rAAV donors with unbiased whole-genome characterization by LOCK-seq.Nucleic acids research · 2026Article
- Metal-Coordinated Histidine-Functionalized Redox-Responsive Polyethyleneimine as a Smart Gene Delivery Vector.Molecular biotechnology · 2026Article
- From delivery to integration: CRISPR-mediated large insert gene editing in primary human T cells.Clinical & translational immunology · 2026Review
- Functional classification of platelet gene variants using CRISPR HDR in CD34American journal of human genetics · 2025Article
- Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia.Molecular therapy. Methods & clinical development · 2025Article
- Machine learning techniques for lipid nanoparticle formulation.Nano convergence · 2025Review
- Gene therapy for sickle cell disease: recent advances, clinical trials and future directions.Cytotherapy · 2025Review
- Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival.Molecular therapy. Nucleic acids · 2025Article
- Genome engineering with Cas9 and AAV repair templates, successes and pitfalls.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Review
- Integration of large genetic payloads using prime editing and site-specific integrases.Nature protocols · 2025Article
- DNA-PKcs inhibition improves sequential gene insertion of the full-lengthMolecular therapy. Nucleic acids · 2024Article
- Enhancement of erythropoietic output by Cas9-mediated insertion of a natural variant in haematopoietic stem and progenitor cells.Nature biomedical engineering · 2024Article
- Emerging Gene-editing nano-therapeutics for Cancer.Heliyon · 2024Review
- Efficient Gene-Editing in Human Pluripotent Stem Cells Through Simplified Assembly of Adeno-Associated Viral (AAV) Donor Templates.Bio-protocol · 2024Article
25 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
The CRISPR/Cas9 system has recently been shown to facilitate high levels of precise genome editing using adeno-associated viral (AAV) vectors to serve as donor template DNA during homologous recombination (HR). However, the maximum AAV packaging capacity of ∼4.5 kb limits the donor size. Here, we overcome this constraint by showing that two co-transduced AAV vectors can serve as donors during consecutive HR events for the integration of large transgenes. Importantly, the method involves a single-step procedure applicable to primary cells with relevance to therapeutic genome editing. We use the methodology in primary human T cells and CD34
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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.