ArticleHuman gene therapy2012
Versatile and efficient genome editing in human cells by combining zinc-finger nucleases with adeno-associated viral vectors.
Article in Human gene therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 32 papers.
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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.
The trial behind it
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
32 citing papers in PubMed, 63 citations in OpenAlex.
- Zinc finger proteins (ZFPs) in health and disease.Molecular biomedicine · 2026Review
- A universal deep-learning model for zinc finger design enables transcription factor reprogramming.Nature biotechnology · 2023Article
- Targeted DNA Demethylation: Vectors, Effectors and Perspectives.Biomedicines · 2023Review
- Improving Homology-Directed Repair in Genome Editing Experiments by Influencing the Cell Cycle.International journal of molecular sciences · 2022Review
- Targeted multi-epitope switching enables straightforward positive/negative selection of CAR T cells.Gene therapy · 2021Article
- A positive, growth-based PAM screen identifies noncanonical motifs recognized by theScience advances · 2020Article
- TraFo-CRISPR: Enhanced Genome Engineering by Transient Foamy Virus Vector-Mediated Delivery of CRISPR/Cas9 Components.Molecular therapy. Nucleic acids · 2019Article
- Genome Editing in Stem Cells for Disease Therapeutics.Molecular biotechnology · 2018Review
- Treatment of hepatitis B virus: an update.Future microbiology · 2016Review
- In Vivo Delivery Systems for Therapeutic Genome Editing.International journal of molecular sciences · 2016Review
- Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes.Molecular therapy. Nucleic acids · 2016Article
- Genome Engineering Using Adeno-associated Virus: Basic and Clinical Research Applications.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- Engineered Viruses as Genome Editing Devices.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- Genome-editing Technologies for Gene and Cell Therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Review
- Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells.Nature protocols · 2015Article
- Recent developments and clinical studies utilizing engineered zinc finger nuclease technology.Cellular and molecular life sciences : CMLS · 2015Review
- Efficient modification of CCR5 in primary human hematopoietic cells using a megaTAL nuclease and AAV donor template.Science translational medicine · 2015Article
- Enhanced gene disruption by programmable nucleases delivered by a minicircle vector.Gene therapy · 2014Article
- Non-integrating gamma-retroviral vectors as a versatile tool for transient zinc-finger nuclease delivery.Scientific reports · 2014Article
- Inverted terminal repeats of adeno-associated virus decrease random integration of a gene targeting fragment in Saccharomyces cerevisiae.BMC molecular biology · 2014Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
9 authors at 1 institution in 1 country.
Funding
Abstract
Zinc-finger nucleases (ZFNs) have become a valuable tool for targeted genome engineering. Based on the enzyme's ability to create a site-specific DNA double-strand break, ZFNs promote genome editing by activating the cellular DNA damage response, including homology-directed repair (HDR) and nonhomologous end-joining. The goal of this study was (i) to demonstrate the versatility of combining the ZFN technology with a vector platform based on adeno-associated virus (AAV), and (ii) to assess the toxicity evoked by this platform. To this end, human cell lines that harbor enhanced green fluorescence protein (EGFP) reporters were generated to easily quantify the frequencies of gene deletion, gene disruption, and gene correction. We demonstrated that ZFN-encoding AAV expression vectors can be employed to induce large chromosomal deletions or to disrupt genes in up to 32% of transduced cells. In combination with AAV vectors that served as HDR donors, the AAV-ZFN platform was utilized to correct a mutation in EGFP in up to 6% of cells. Genome editing on the DNA level was confirmed by genotyping. Although cell cycle profiling revealed a modest G2/M arrest at high AAV-ZFN vector doses, platform-induced apoptosis could not be detected. In conclusion, the combined AAV-ZFN vector technology is a useful tool to edit the human genome with high efficiency. Because AAV vectors can transduce many cell types relevant for gene therapy, the ex vivo and in vivo delivery of ZFNs via AAV vectors will be of great interest for the treatment of inherited disorders.
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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.