ArticleMolecular therapy. Methods & clinical development2020
A Single "All-in-One" Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair.
Article in Molecular therapy. Methods & clinical development, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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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.
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Who cites it
21 citing papers in PubMed.
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Prospects of viral vector-mediated delivery of sequences encoding anti-HBV designer endonucleases.Gene therapy · 2025Review
- Emerging Gene-editing nano-therapeutics for Cancer.Heliyon · 2024Review
- Current developments of gene therapy in human diseases.MedComm · 2024Review
- Construction and Stability of All-in-One Adenovirus Vectors Simultaneously Expressing Four and Eight Multiplex Guide RNAs and Cas9 Nickase.International journal of molecular sciences · 2024Article
- Construction and application of adenoviral vectors.Molecular therapy. Nucleic acids · 2023Review
- Targeted adenovirus-mediated transduction of human T cellsMolecular therapy. Methods & clinical development · 2023Article
- Progress in Respiratory Gene Therapy.Human gene therapy · 2022Review
- Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells.Nucleic acids research · 2022Article
- Anticipating New Treatments for Cystic Fibrosis: A Global Survey of Researchers.Journal of clinical medicine · 2022Article
- The use of adenoviral vectors in gene therapy and vaccine approaches.Genetics and molecular biology · 2022Article
- Advances in CRISPR/Cas9.BioMed research international · 2022Review
- Novel vectors and approaches for gene therapy in liver diseases.JHEP reports : innovation in hepatology · 2021Review
- Exosome/Liposome-like Nanoparticles: New Carriers for CRISPR Genome Editing in Plants.International journal of molecular sciences · 2021Review
- iMATCH: an integrated modular assembly system for therapeutic combination high-capacity adenovirus gene therapy.Molecular therapy. Methods & clinical development · 2021Article
- Gene Therapy: A Possible Alternative to CFTR Modulators?Frontiers in pharmacology · 2021Article
- Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.Journal of controlled release : official journal of the Controlled Release Society · 2020Review
- New Directions in Pulmonary Gene Therapy.Human gene therapy · 2020Review
- Treating Cystic Fibrosis with mRNA and CRISPR.Human gene therapy · 2020Review
- Review
Corrections and comments
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Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In this study, we developed a single helper-dependent adenovirus (HDAd) to deliver all of the components (donor DNA, CRISPR-associated protein 9 [Cas9], and guide RNA [gRNA]) needed to achieve high-efficiency gene targeting and homology-directed repair in transduced cells. We show that these "all-in-one" HDAds are up to 117-fold more efficient at gene targeting than donor HDAds that do not express CRISPR/Cas9 in human induced pluripotent stem cells (iPSCs). The vast majority (>90%) of targeted recombinants had only one allele targeted, and this was accompanied by high-frequency indel formation in the non-targeted allele at the site of Cas9 cleavage. These indels varied in size and nature, and included large deletions of ∼8 kb. The remaining minority of recombinants had both alleles targeted (so-called bi-allelic targeting). These all-in-one HDAds represent an important platform for accomplishing and expanding the utility of homology-directed repair, especially for difficult-to-transfect cells and for
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Registered trials
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