ArticleMolecular therapy : the journal of the American Society of Gene Therapy2009
Regulated and multiple miRNA and siRNA delivery into primary cells by a lentiviral platform.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 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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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
45 citing papers in PubMed.
- YTHDC1-mediated microRNA maturation is essential for hematopoietic stem cells maintenance.Cell death discovery · 2024Article
- From Crypts to Cancer: A Holistic Perspective on Colorectal Carcinogenesis and Therapeutic Strategies.International journal of molecular sciences · 2024Review
- Article
- Efficient shRNA-based knockdown of multiple target genes for cell therapy using a chimeric miRNA cluster platform.Molecular therapy. Nucleic acids · 2023Article
- The transcription factor HIF2α partakes in the differentiation block of acute myeloid leukemia.EMBO molecular medicine · 2023Article
- Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.Molecular therapy. Nucleic acids · 2023Article
- Identification of the global miR-130a targetome reveals a role for TBL1XR1 in hematopoietic stem cell self-renewal and t(8;21) AML.Cell reports · 2022Article
- IPEX Syndrome: Improved Knowledge of Immune Pathogenesis Empowers Diagnosis.Frontiers in pediatrics · 2021Review
- GATA2 controls lymphatic endothelial cell junctional integrity and lymphovenous valve morphogenesis throughDevelopment (Cambridge, England) · 2019Article
- MicroRNA-127-3p controls murine hematopoietic stem cell maintenance by limiting differentiation.Haematologica · 2019Article
- Overexpression of MicroRNA-148b-3p stimulates osteogenesis of human bone marrow-derived mesenchymal stem cells: the role of MicroRNA-148b-3p in osteogenesis.BMC medical genetics · 2019Article
- Targeting regulatory T cells for improving cancer therapy: Challenges and prospects.Cancer reports (Hoboken, N.J.) · 2018Review
- miR-21 depletion in macrophages promotes tumoricidal polarization and enhances PD-1 immunotherapy.Oncogene · 2018Article
- Therapeutic Potential of Gene-Modified Regulatory T Cells: From Bench to Bedside.Frontiers in immunology · 2018Review
- Anti-leukemic activity of microRNA-26a in a chronic lymphocytic leukemia mouse model.Oncogene · 2017Article
- Preclinical Evaluation of a Lentiviral Vector for Huntingtin Silencing.Molecular therapy. Methods & clinical development · 2017Article
- A Lentiviral Fluorescent Genetic Barcoding System for Flow Cytometry-Based Multiplex Tracking.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Engineered Tolerance: Tailoring Development, Function, and Antigen-Specificity of Regulatory T Cells.Frontiers in immunology · 2017Review
- Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype.The Journal of clinical investigation · 2016Article
- Leukocytes recruited by tumor-derived HMGB1 sustain peritoneal carcinomatosis.Oncoimmunology · 2016Article
Corrections and comments
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Authors and funding
6 authors.
Funding
Abstract
RNA interference (RNAi) has tremendous potential for investigating gene function and developing new therapies. However, the design and validation of proficient vehicles for stable and safe microRNA (miR) and small interfering RNA (siRNA) delivery into relevant target cells remains an active area of investigation. Here, we developed a lentiviral platform to efficiently coexpress one or more natural/artificial miR together with a gene of interest from constitutive or regulated polymerase-II (Pol-II) promoters. By swapping the stem-loop (sl) sequence of a selected primary transcript (pri-miR) with that of other miR or replacing the stem with an siRNA of choice, we consistently obtained robust expression of the chimeric/artificial miR in several cell types. We validated our platform transducing a panel of engineered cells stably expressing sensitive reporters for miR activity and on a natural target. This approach allowed us to quantitatively assess at steady state the target suppression activity and expression level of each delivered miR and to compare it to those of endogenous miR. Exogenous/artificial miR reached the concentration and activity typical of highly expressed natural miR without perturbing endogenous miR maturation or regulation. Finally, we demonstrate the robust performance of the platform reversing the anergic/suppressive phenotype of human primary regulatory T cells (Treg) by knocking-down their master gene Forkhead Transcription Factor P3 (FOXP3).
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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.