ArticleMolecular therapy : the journal of the American Society of Gene Therapy2020
Efficient Nuclease-Directed Integration of Lentivirus Vectors into the Human Ribosomal DNA Locus.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2020. 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, 29 citations in OpenAlex.
- Optimized R2 retroelement complexes for DNA insertion into plant genomes.Nature biotechnology · 2026Article
- A targeting lentiviral vector for generation of CAR-T cells in vivo.Scientific reports · 2026Article
- Adaptation of lentiviral vectors for viral gene therapy and their impact on host cell biology.Journal of translational medicine · 2026Review
- Rewriting the genome: harnessing R2 retrotransposons for precise DNA insertion.Frontiers in genome editing · 2026Review
- Application of biomimetic approaches in the treatment of neurological disorders.Materials today. Bio · 2025Review
- Long Non-coding RNA Based Therapy for Cardiovascular Disease.Journal of cardiovascular translational research · 2025Review
- Biology and utilization of R2 retrotransposons.RNA biology · 2025Review
- Gene-sized DNA insertion at genomic safe harbors in human cells using a site-directed transposase.Nucleic acids research · 2025Article
- Precise progerin targeting using RfxCas13d: A therapeutic avenue for Hutchinson-Gilford progeria syndrome.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Ribosome biogenesis: A central player in liver diseases.Genes & diseases · 2025Review
- Structure and biochemistry-guided engineering of an all-RNA system for DNA insertion with R2 retrotransposons.Nature communications · 2025Article
- In vivo placental gene modulation via sonoporation.Placenta · 2025Article
- Harnessing eukaryotic retroelement proteins for transgene insertion into human safe-harbor loci.Nature biotechnology · 2025Article
- Gene therapy and kidney diseases.Molecular therapy. Methods & clinical development · 2024Review
- Approaches and applications in transdermal and transpulmonary gene drug delivery.Frontiers in bioengineering and biotechnology · 2024Review
- Ethical implications of developing RNA-based therapies for cardiovascular disorders.Frontiers in bioengineering and biotechnology · 2024Review
- Advancing breast cancer diagnosis with a near-infrared fluorescence imaging smart sensor for estrogen/progesterone receptor detection.Scientific reports · 2023Article
- Human papillomavirus in the setting of immunodeficiency: Pathogenesis and the emergence of next-generation therapies to reduce the high associated cancer risk.Frontiers in immunology · 2023Review
- Article
- Targeted Nanocarrier Delivery of RNA Therapeutics to Control HIV Infection.Pharmaceutics · 2022Review
Corrections and comments
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Authors and funding
5 authors at 3 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Lentivirus vectors (LVs) are efficient tools for gene transfer, but the non-specific nature of transgene integration by the viral integration machinery carries an inherent risk for genotoxicity. We modified the integration machinery of LVs and harnessed the cellular DNA double-strand break repair machinery to integrate transgenes into ribosomal DNA, a promising genomic safe-harbor site for transgenes. LVs carrying modified I-PpoI-derived homing endonuclease proteins were characterized in detail, and we found that at least 21% of all integration sites localized to ribosomal DNA when LV transduction was coupled to target DNA cleavage. In addition to the primary sequence recognized by the endonuclease, integration was also enriched in chromatin domains topologically associated with nucleoli, which contain the targeted ribosome RNA genes. Targeting of this highly repetitive region for integration was not associated with detectable DNA deletions or negative impacts on cell health in transduced primary human T cells. The modified LVs characterized here have an overall lower risk for insertional mutagenesis than regular LVs and can thus improve the safety of gene and cellular therapy.
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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.