ArticleHuman gene therapy methods2013
Enhancing the utility of adeno-associated virus gene transfer through inducible tissue-specific expression.
Article in Human gene therapy methods, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 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
15 citing papers in PubMed.
- Gene Therapy for Acquired and Genetic Cholestasis.Biomedicines · 2022Review
- Membrane-bound MMP-14 protease-activatable adeno-associated viral vectors for gene delivery to pancreatic tumors.Gene therapy · 2022Article
- Strategies to improve safety profile of AAV vectors.Frontiers in molecular medicine · 2022Review
- Advances in the Development and the Applications of Nonviral, Episomal Vectors for Gene Therapy.Human gene therapy · 2021Article
- Tissue and cell-type-specific transduction using rAAV vectors in lung diseases.Journal of molecular medicine (Berlin, Germany) · 2021Review
- Reducing off target viral delivery in ovarian cancer gene therapy using a protease-activated AAV2 vector platform.Journal of controlled release : official journal of the Controlled Release Society · 2019Article
- mRNA-Based Protein Replacement Therapy for the Heart.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Review
- AAVvector-mediated in vivo reprogramming into pluripotency.Nature communications · 2018Article
- Next-generation AAV vectors for clinical use: an ever-accelerating race.Virus genes · 2017Review
- Overexpression and deletion of phospholipid transfer protein reduce HDL mass and cholesterol efflux capacity but not macrophage reverse cholesterol transport.Journal of lipid research · 2017Article
- Downregulation of the β1 adrenergic receptor in the myocardium results in insensitivity to metoprolol and reduces blood pressure in spontaneously hypertensive rats.Molecular medicine reports · 2017Article
- Identification and Validation of Small Molecules That Enhance Recombinant Adeno-associated Virus Transduction following High-Throughput Screens.Journal of virology · 2016Article
- Gene therapy for the treatment of heart failure: promise postponed.European heart journal · 2016Review
- Riboswitch-mediated Attenuation of Transgene Cytotoxicity Increases Adeno-associated Virus Vector Yields in HEK-293 Cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2015Article
- Synthetic chemically modified mRNA (modRNA): toward a new technology platform for cardiovascular biology and medicine.Cold Spring Harbor perspectives in medicine · 2014Review
Corrections and comments
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Authors and funding
8 authors.
Funding
Abstract
The ability to regulate both the timing and specificity of gene expression mediated by viral vectors will be important in maximizing its utility. We describe the development of an adeno-associated virus (AAV)-based vector with tissue-specific gene regulation, using the ARGENT dimerizer-inducible system. This two-vector system based on AAV serotype 9 consists of one vector encoding a combination of reporter genes from which expression is directed by a ubiquitous, inducible promoter and a second vector encoding transcription factor domains under the control of either a heart- or liver-specific promoter, which are activated with a small molecule. Administration of the vectors via either systemic or intrapericardial injection demonstrated that the vector system is capable of mediating gene expression that is tissue specific, regulatable, and reproducible over induction cycles. Somatic gene transfer in vivo is being considered in therapeutic applications, although its most substantial value will be in basic applications such as target validation and development of animal models.
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Registered trials
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