ArticleNucleic acids research2009
Efficient targeting of a SCID gene by an engineered single-chain homing endonuclease.
Article in Nucleic acids research, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 81 papers.
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Who cites it
81 citing papers in PubMed.
- AI-driven advances in plant biotechnology: sharpening the edge of plant tissue culture and genome editing.Frontiers in plant science · 2025Review
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- Compact zinc finger architecture utilizing toxin-derived cytidine deaminases for highly efficient base editing in human cells.Nature communications · 2024Article
- Advancements in genome editing tools for genetic studies and crop improvement.Frontiers in plant science · 2024Review
- Rare immune diseases paving the road for genome editing-based precision medicine.Frontiers in genome editing · 2023Review
- Unclasping potentials of genomics and gene editing in chickpea to fight climate change and global hunger threat.Frontiers in genetics · 2023Review
- "Cutting the Mustard" with Induced Pluripotent Stem Cells: An Overview and Applications in Healthcare Paradigm.Stem cell reviews and reports · 2022Review
- Genome editing via non-viral delivery platforms: current progress in personalized cancer therapy.Molecular cancer · 2022Review
- Comparison of the Feasibility, Efficiency, and Safety of Genome Editing Technologies.International journal of molecular sciences · 2021Review
- Gene Editing in Pluripotent Stem Cells and Their Derived Organoids.Stem cells international · 2021Review
- Enhancing gene editing specificity by attenuating DNA cleavage kinetics.Nature biotechnology · 2019Article
- Active site residue identity regulates cleavage preference of LAGLIDADG homing endonucleases.Nucleic acids research · 2018Article
- Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol.Nature biotechnology · 2018Article
- Application of Genome Editing Techniques in Immunology.Archivum immunologiae et therapiae experimentalis · 2018Review
- Engineering altered protein-DNA recognition specificity.Nucleic acids research · 2018Review
- CRISPR-Cas9 Genome Editing for Treatment of Atherogenic Dyslipidemia.Arteriosclerosis, thrombosis, and vascular biology · 2018Review
- Crystallographic analyses illustrate significant plasticity and efficient recoding of meganuclease target specificity.Nucleic acids research · 2017Article
- A history of genome editing in mammals.Mammalian genome : official journal of the International Mammalian Genome Society · 2017Review
- Integration of a CD19 CAR into the TCR Alpha Chain Locus Streamlines Production of Allogeneic Gene-Edited CAR T Cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
21 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
13 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sequence-specific endonucleases recognizing long target sequences are emerging as powerful tools for genome engineering. These endonucleases could be used to correct deleterious mutations or to inactivate viruses, in a new approach to molecular medicine. However, such applications are highly demanding in terms of safety. Mutations in the human RAG1 gene cause severe combined immunodeficiency (SCID). Using the I-CreI dimeric LAGLIDADG meganuclease as a scaffold, we describe here the engineering of a series of endonucleases cleaving the human RAG1 gene, including obligate heterodimers and single-chain molecules. We show that a novel single-chain design, in which two different monomers are linked to form a single molecule, can induce high levels of recombination while safeguarding more effectively against potential genotoxicity. We provide here the first demonstration that an engineered meganuclease can induce targeted recombination at an endogenous locus in up to 6% of transfected human cells. These properties rank this new generation of endonucleases among the best molecular scissors available for genome surgery strategies, potentially avoiding the deleterious effects of previous gene therapy approaches.
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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.