ArticleMolecular therapy : the journal of the American Society of Gene Therapy2012
Efficient and accurate homologous recombination in hESCs and hiPSCs using helper-dependent adenoviral vectors.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.
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Who cites it
28 citing papers in PubMed, 47 citations in OpenAlex.
- Review
- Genetic modification by overexpression of target gene in mesenchymal stromal cell for treating liver diseases.Journal of molecular medicine (Berlin, Germany) · 2021Review
- A Single "All-in-One" Helper-Dependent Adenovirus to Deliver Donor DNA and CRISPR/Cas9 for Efficient Homology-Directed Repair.Molecular therapy. Methods & clinical development · 2020Article
- Review
- Bi-allelic Homology-Directed Repair with Helper-Dependent Adenoviruses.Molecular therapy. Methods & clinical development · 2019Article
- Dual usage of a stage-specific fluorescent reporter system based on a helper-dependent adenoviral vector to visualize osteogenic differentiation.Scientific reports · 2019Article
- Production of CRISPR/Cas9-Mediated Self-Cleaving Helper-Dependent Adenoviruses.Molecular therapy. Methods & clinical development · 2019Article
- Transcriptional profiling of isogenic Friedreich ataxia neurons and effect of an HDAC inhibitor on disease signatures.The Journal of biological chemistry · 2019Article
- Gene Editing with Helper-Dependent Adenovirus Can Efficiently Introduce Multiple Changes Simultaneously over a Large Genomic Region.Molecular therapy. Nucleic acids · 2017Article
- Simple Derivation of Spinal Motor Neurons from ESCs/iPSCs Using Sendai Virus Vectors.Molecular therapy. Methods & clinical development · 2017Article
- Integration-defective lentiviral vector mediates efficient gene editing through homology-directed repair in human embryonic stem cells.Nucleic acids research · 2017Article
- An inhibitor of fibroblast growth factor receptor-1 (FGFR1) promotes late-stage terminal differentiation from NGN3+ pancreatic endocrine progenitors.Scientific reports · 2016Article
- Homology Requirements for Efficient, Footprintless Gene Editing at the CFTR Locus in Human iPSCs with Helper-dependent Adenoviral Vectors.Molecular therapy. Nucleic acids · 2016Article
- Embryonic type NaScientific reports · 2016Article
- Comprehensive Protocols for CRISPR/Cas9-based Gene Editing in Human Pluripotent Stem Cells.Current protocols in stem cell biology · 2016Article
- Human induced pluripotent stem cells for monogenic disease modelling and therapy.World journal of stem cells · 2016Review
- Modulation of human allogeneic and syngeneic pluripotent stem cells and immunological implications for transplantation.Transplantation reviews (Orlando, Fla.) · 2016Review
- Engineered Viruses as Genome Editing Devices.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- BTK gene targeting by homologous recombination using a helper-dependent adenovirus/adeno-associated virus hybrid vector.Gene therapy · 2016Article
- Human Induced Pluripotent Stem Cell NEUROG2 Dual Knockin Reporter Lines Generated by the CRISPR/Cas9 System.Stem cells and development · 2015Article
Corrections and comments
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Authors and funding
13 authors at 3 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Low efficiencies of gene targeting via homologous recombination (HR) have limited basic research and applications using human embryonic stem cells (hESCs) and human induced pluripotent stem cells (hiPSCs). Here, we show highly and equally efficient gene knockout and knock-in at both transcriptionally active (HPRT1, KU80, LIG1, LIG3) and inactive (HB9) loci in these cells using high-capacity helper-dependent adenoviral vectors (HDAdVs). Without the necessity of introducing artificial DNA double-strand breaks, 7-81% of drug-resistant colonies were gene-targeted by accurate HR, which were not accompanied with additional ectopic integrations. Even at the motor neuron-specific HB9 locus, the enhanced green fluorescent protein (EGFP) gene was accurately knocked in in 23-57% of drug-resistant colonies. In these clones, induced differentiation into the HB9-positive motor neuron correlated with EGFP expression. Furthermore, HDAdV infection had no detectable adverse effects on the undifferentiated state and pluripotency of hESCs and hiPSCs. These results suggest that HDAdV is one of the best methods for efficient and accurate gene targeting in hESCs and hiPSCs and might be especially useful for therapeutic 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.