ArticleHuman molecular genetics2017
Quantitative assessment of timing, efficiency, specificity and genetic mosaicism of CRISPR/Cas9-mediated gene editing of hemoglobin beta gene in rhesus monkey embryos.
Article in Human molecular genetics, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 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.
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Who cites it
17 citing papers in PubMed, 46 citations in OpenAlex.
- Article
- The history, use, and challenges of therapeutic somatic cell and germline gene editing.Fertility and sterility · 2023Review
- CATI: an efficient gene integration method for rodent and primate embryos by MMEJ suppression.Genome biology · 2023Article
- CRISPR/Cas9 system: a reliable and facile genome editing tool in modern biology.Molecular biology reports · 2022Review
- CRISPR/Cas9 genome editing to create nonhuman primate models for studying stem cell therapies for HIV infection.Retrovirology · 2022Review
- Modeling genetic diseases in nonhuman primates through embryonic and germline modification: Considerations and challenges.Science translational medicine · 2022Review
- Gene editing monkeys: Retrospect and outlook.Frontiers in cell and developmental biology · 2022Review
- Whole genome sequencing ofFrontiers in genome editing · 2022Article
- Non-human primates as a model for human development.Stem cell reports · 2021Review
- Development of CRISPR technology for precise single-base genome editing: a brief review.BMB reports · 2021Review
- The dawn of non-human primate models for neurodevelopmental disorders.Current opinion in genetics & development · 2020Review
- Genome editing of CCR5 by CRISPR-Cas9 in Mauritian cynomolgus macaque embryos.Scientific reports · 2020Article
- CRISPR/Cas9-mediated genome editing in nonhuman primates.Disease models & mechanisms · 2019Review
- Targeted Gene Disruption in Pacific Oyster Based on CRISPR/Cas9 Ribonucleoprotein Complexes.Marine biotechnology (New York, N.Y.) · 2019Article
- Deriving rabbit embryonic stem cells by small molecule inhibitors.American journal of translational research · 2019Article
- Identification of on-target mutagenesis during correction of a beta-thalassemia splice mutation in iPS cells with optimised CRISPR/Cas9-double nickase reveals potential safety concerns.APL bioengineering · 2018Article
- Mosaicism diminishes the value of pre-implantation embryo biopsies for detecting CRISPR/Cas9 induced mutations in sheep.Transgenic research · 2018Article
Corrections and comments
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Authors and funding
9 authors at 3 institutions in 1 country.
Funding
Abstract
Gene editing technologies offer new options for developing novel biomedical research models and for gene and stem cell based therapies. However, applications in many species demand high efficiencies, specificity, and a thorough understanding of likely editing outcomes. To date, overall efficiencies, rates of off-targeting and degree of genetic mosaicism have not been well-characterized for most species, limiting our ability to optimize methods. As a model gene for measuring these parameters of the CRISPR/Cas9 application in a primate species (rhesus monkey), we selected the β-hemoglobin gene (HBB), which also has high relevance to the potential application of gene editing and stem-cell technologies for treating human disease. Our data demonstrate an ability to achieve a high efficiency of gene editing in rhesus monkey zygotes, with no detected off-target effects at selected off-target loci. Considerable genetic mosaicism and variation in the fraction of embryonic cells bearing targeted alleles are observed, and the timing of editing events is revealed using a new model. The uses of Cas9-WT protein combined with optimized concentrations of sgRNAs are two likely areas for further refinement to enhance efficiency while limiting unfavorable outcomes that can be exceedingly costly for application of gene editing in primate species.
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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.