ArticleGene therapy2016
CRISPR-on system for the activation of the endogenous human INS gene.
Article in Gene therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
What it found
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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
21 citing papers in PubMed, 52 citations in OpenAlex.
- Role of hydroxymethylglutharyl-coenzyme A reductase in the induction of stem-like states in breast cancer.Journal of cancer research and clinical oncology · 2024Article
- Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine.Frontiers in genome editing · 2024Review
- STAT1 is required to establish but not maintain interferon-γ-induced transcriptional memory.The EMBO journal · 2023Article
- CRISPR-Cas-mediated transcriptional modulation: The therapeutic promises of CRISPRa and CRISPRi.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- Activation of Insulin Gene Expression via Transfection of a CRISPR/dCas9a System Using Magnetic Peptide-Imprinted Nanoparticles.Pharmaceutics · 2023Article
- Advances in CRISPR therapeutics.Nature reviews. Nephrology · 2023Review
- Current advances and future prospects in production of recombinant insulin and other proteins to treat diabetes mellitus.Biotechnology letters · 2022Review
- Genome Editing Using CRISPR-Cas9 and Autoimmune Diseases: A Comprehensive Review.International journal of molecular sciences · 2022Review
- Cancer/Testis Antigens as Biomarker and Target for the Diagnosis, Prognosis, and Therapy of Lung Cancer.Frontiers in oncology · 2022Review
- Reprogramming the anti-tumor immune response via CRISPR genetic and epigenetic editing.Molecular therapy. Methods & clinical development · 2021Review
- Approaching Shared Pathophysiology in Immune-Mediated Diseases through Functional Genomics.Genes · 2020Review
- From Mesenchymal Stromal/Stem Cells to Insulin-Producing Cells: Progress and Challenges.Stem cell reviews and reports · 2020Review
- Functional Genomics in Pancreatic β Cells: Recent Advances in Gene Deletion and Genome Editing Technologies for Diabetes Research.Frontiers in endocrinology · 2020Review
- Dead Cas Systems: Types, Principles, and Applications.International journal of molecular sciences · 2019Review
- Tumour suppression by targeted intravenous non-viral CRISPRa using dendritic polymers.Chemical science · 2019Article
- Therapies for Type 1 Diabetes: Current Scenario and Future Perspectives.Clinical medicine insights. Endocrinology and diabetes · 2019Review
- Delivery systems of CRISPR/Cas9-based cancer gene therapy.Journal of biological engineering · 2018Review
- Gene Editing and Human Pluripotent Stem Cells: Tools for Advancing Diabetes Disease Modeling and Beta-Cell Development.Current diabetes reports · 2017Review
- CRISPR-targeted genome editing of mesenchymal stem cell-derived therapies for type 1 diabetes: a path to clinical success?Stem cell research & therapy · 2017Review
- The Nexus of Stem Cell-Derived Beta-Cells and Genome Engineering.The review of diabetic studies : RDS · 2017Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Advances in the field of epigenetics have allowed the design of new therapeutic strategies to address complex diseases such as type 1 diabetes (T1D). Clustered regularly interspaced short palindromic repeats (CRISPR)-on is a novel and powerful RNA-guided transcriptional activator system that can turn on specific gene expression; however, it remains unclear whether this system can be widely used or whether its use will be restricted depending on cell types, methylation promoter statuses or the capacity to modulate chromatin state. Our results revealed that the CRISPR-on system fused with transcriptional activators (dCas9-VP160) activated endogenous human INS, which is a silenced gene with a fully methylated promoter. Similarly, we observed a synergistic effect on gene activation when multiple single guide RNAs were used, and the transcriptional activation was maintained until day 21. Regarding the epigenetic profile, the targeted promoter gene did not exhibit alteration in its methylation status but rather exhibited altered levels of H3K9ac following treatment. Importantly, we showed that dCas9-VP160 acts on patients' cells in vitro, particularly the fibroblasts of patients with T1D.
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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.