ArticleHuman gene therapy2017
A 5' Noncoding Exon Containing Engineered Intron Enhances Transgene Expression from Recombinant AAV Vectors in vivo.
Article in Human gene therapy, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers, 1 of them a synthesis that pooled it.
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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
15 citing papers in PubMed, 1 synthesis or guideline pooled it, 25 citations in OpenAlex.
- Recombinant Adeno-Associated Viral Vectors (rAAV)-Vector Elements in Ocular Gene Therapy Clinical Trials and Transgene Expression and Bioactivity Assays.International journal of molecular sciences · 2020Pooled it
- AAV vector development, back to the future.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Adeno-associated virus as a delivery vector for gene therapy of human diseases.Signal transduction and targeted therapy · 2024Review
- Selection of viral capsids and promoters affects the efficacy of rescue ofMolecular therapy. Methods & clinical development · 2023Article
- Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors.Molecular therapy. Nucleic acids · 2023Review
- Improving adeno-associated viral (AAV) vector-mediated transgene expression in retinal ganglion cells: comparison of five promoters.Gene therapy · 2023Article
- The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner.Nature communications · 2023Article
- Intravitreal Injection of AAV for the Transduction of Mouse Retinal Ganglion Cells.Methods in molecular biology (Clifton, N.J.) · 2023Article
- AAV vectors: The Rubik's cube of human gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- Fludarabine increases nuclease-free AAV- and CRISPR/Cas9-mediated homologous recombination in mice.Nature biotechnology · 2022Article
- Optimization of miR-22 expression cassette for rAAV delivery on diabetes.Molecular biomedicine · 2022Article
- Progress of Transposon Vector System for Production of Recombinant Therapeutic Proteins in Mammalian Cells.Frontiers in bioengineering and biotechnology · 2022Review
- Adeno-associated virus vector as a platform for gene therapy delivery.Nature reviews. Drug discovery · 2019Review
- Exploring the role of NCCR variation on JC polyomavirus expression from dual reporter minicircles.PloS one · 2018Article
- Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.Journal of inherited metabolic disease · 2017Review
Corrections and comments
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Authors and funding
6 authors at 2 institutions in 1 country.
Funding
Abstract
We previously developed a mini-intronic plasmid (MIP) expression system in which the essential bacterial elements for plasmid replication and selection are placed within an engineered intron contained within a universal 5' UTR noncoding exon. Like minicircle DNA plasmids (devoid of bacterial backbone sequences), MIP plasmids overcome transcriptional silencing of the transgene. However, in addition MIP plasmids increase transgene expression by 2 and often >10 times higher than minicircle vectors in vivo and in vitro. Based on these findings, we examined the effects of the MIP intronic sequences in a recombinant adeno-associated virus (AAV) vector system. Recombinant AAV vectors containing an intron with a bacterial replication origin and bacterial selectable marker increased transgene expression by 40 to 100 times in vivo when compared with conventional AAV vectors. Therefore, inclusion of this noncoding exon/intron sequence upstream of the coding region can substantially enhance AAV-mediated gene expression in vivo.
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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.