ArticleMolecular therapy. Nucleic acids2013
Adeno-associated Virus-mediated, Mifepristone-regulated Transgene Expression in the Brain.
Article in Molecular therapy. Nucleic acids, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.
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Who cites it
16 citing papers in PubMed, 30 citations in OpenAlex.
- Adeno-associated virus vector delivery to the brain: Technology advancements and clinical applications.Advanced drug delivery reviews · 2024Review
- All-in-one IQ toggle switches with high versatilities for fine-tuning of transgene expression in mammalian cells and tissues.Molecular therapy. Methods & clinical development · 2024Article
- Lentiviral vectors for inducible, transactivator-free advanced therapy medicinal products: Application to CAR-T cells.Molecular therapy. Nucleic acids · 2023Article
- Optimized pharmacological control over the AAV-Gene-Switch vector for regulable gene therapy.Molecular therapy. Methods & clinical development · 2021Article
- Externally-Controlled Systems for Immunotherapy: From Bench to Bedside.Frontiers in immunology · 2020Review
- In-Depth Characterization of a Mifepristone-Regulated Expression System for AAV5-Mediated Gene Therapy in the Liver.Molecular therapy. Methods & clinical development · 2019Article
- Biological, Behavioral and Physiological Consequences of Drug-Induced Pregnancy Termination at First-Trimester Human Equivalent in an Animal Model.Frontiers in neuroscience · 2019Article
- A MicroRNA124 Target Sequence Restores Astrocyte Specificity of gfaABCMolecular therapy. Nucleic acids · 2017Article
- Review
- Current Experimental Studies of Gene Therapy in Parkinson's Disease.Frontiers in aging neuroscience · 2017Review
- Ecdysone Receptor-based Singular Gene Switches for Regulated Transgene Expression in Cells and Adult Rodent Tissues.Molecular therapy. Nucleic acids · 2016Article
- Mifepristone-inducible transgene expression in neural progenitor cells in vitro and in vivo.Gene therapy · 2016Article
- Tet-On Systems For Doxycycline-inducible Gene Expression.Current gene therapy · 2016Review
- A regulatable AAV vector mediating GDNF biological effects at clinically-approved sub-antimicrobial doxycycline doses.Molecular therapy. Methods & clinical development · 2016Article
- Neuronal expression of the human neuropeptide S receptor NPSR1 identifies NPS-induced calcium signaling pathways.PloS one · 2015Article
- Synthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseases.BioEssays : news and reviews in molecular, cellular and developmental biology · 2014Review
Corrections and comments
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Authors and funding
4 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Gene therapy, in its current configuration, is irreversible and does not allow control over transgene expression in case of side effects. Only few regulated vector systems are available, and none of these has reached clinical applicability yet. The mifepristone (Mfp)-regulated Gene Switch (GS) system is characterized by promising features such as being composed of mainly human components and an approved small-molecule drug as an inducer. However, it has not yet been evaluated in adeno-associated virus (AAV) vectors, neither has it been tested for applicability in viral vectors in the central nervous system (CNS). Here, we demonstrate that the GS system can be used successfully in AAV vectors in the brain, and that short-term induced glial cell line-derived neurotrophic factor (GDNF) expression prevented neurodegeneration in a rodent model of Parkinson's disease (PD). We also demonstrate repeated responsiveness to the inducer Mfp and absence of immunological tissue reactions in the rat brain. Human equivalent dosages of Mfp used in this study were lower than those used safely for treatment of psychiatric threats, indicating that the inducer could be safely applied in patients. Our results suggest that the GS system in AAV vectors is well suited for further development towards clinical applicability.Molecular Therapy-Nucleic Acids (2013) 2, e106; doi:10.1038/mtna.2013.35; published online 16 July 2013.
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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.