ArticleCell research2019
Precise in vivo genome editing via single homology arm donor mediated intron-targeting gene integration for genetic disease correction.
Article in Cell research, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 48 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.
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Who cites it
48 citing papers in PubMed, 67 citations in OpenAlex.
- Loss of ABCA4 from photoreceptor discs is associated with glial transcriptomic changes in retinal organoids.Stem cells (Dayton, Ohio) · 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
- Directed evolution on compact landing pads yields highly efficient recombinases for large DNA integration.Nucleic acids research · 2026Article
- Advancements in CRISPR-basedFrontiers in genome editing · 2026Review
- Targeted in vivo gene integration of a secretion-enabled GLP-1 receptor agonist reverses diet-induced non-genetic obesity and pre-diabetes.Communications medicine · 2025Article
- Gene therapy shines light on congenital stationary night blindness for future cures.Journal of translational medicine · 2025Review
- Beyond a few bases: methods for large DNA insertion and gene targeting in plants.The Plant journal : for cell and molecular biology · 2025Review
- Development of efficient targeted insertion mediated by CRISPR-Cas12a and homology-directed repair in maize.Frontiers in genome editing · 2025Article
- Emerging tools in plant genome editing.Frontiers in genome editing · 2025Review
- Exploration of the potential of genomic editing in the treatment of congenital adrenal hyperplasia.Frontiers in endocrinology · 2025Review
- Gene Editing for Duchenne Muscular Dystrophy: From Experimental Models to Emerging Therapies.Degenerative neurological and neuromuscular disease · 2025Review
- Lipid Nanoparticles Enable Efficient In Vivo DNA Knock-In via HITI-Mediated Genome Editing.Biomolecules · 2024Article
- GEARBOCS: An Adeno Associated Virus Tool forbioRxiv : the preprint server for biology · 2024Article
- REMOVER-PITCh: microhomology-assisted long-range gene replacement with highly multiplexed CRISPR-Cas9.In vitro cellular & developmental biology. Animal · 2024Article
- Therapeutic strategy for spinal muscular atrophy by combining gene supplementation and genome editing.Nature communications · 2024Article
- Genome editing and kidney health.Clinical kidney journal · 2024Review
- Single-cell synaptome mapping: its technical basis and applications in critical period plasticity research.Frontiers in neural circuits · 2024Review
- Article
- Superior Fidelity and Distinct Editing Outcomes of SaCas9 Compared with SpCas9 in Genome Editing.Genomics, proteomics & bioinformatics · 2023Article
- Recent advances and challenges in the use of CRISPR/Cas9 genome editing for understanding neuronal cell biology.Neurophotonics · 2023Article
Corrections and comments
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Authors and funding
27 authors at 8 institutions in 5 countries.
Funding
Abstract
In vivo genome editing represents a powerful strategy for both understanding basic biology and treating inherited diseases. However, it remains a challenge to develop universal and efficient in vivo genome-editing tools for tissues that comprise diverse cell types in either a dividing or non-dividing state. Here, we describe a versatile in vivo gene knock-in methodology that enables the targeting of a broad range of mutations and cell types through the insertion of a minigene at an intron of the target gene locus using an intracellularly linearized single homology arm donor. As a proof-of-concept, we focused on a mouse model of premature-aging caused by a dominant point mutation, which is difficult to repair using existing in vivo genome-editing tools. Systemic treatment using our new method ameliorated aging-associated phenotypes and extended animal lifespan, thus highlighting the potential of this methodology for a broad range of in vivo genome-editing applications.
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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.