ArticleMolecular therapy. Methods & clinical development2018
Destabilizing Domains Enable Long-Term and Inert Regulation of GDNF Expression in the Brain.
Article in Molecular therapy. Methods & clinical development, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
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Who cites it
14 citing papers in PubMed, 19 citations in OpenAlex.
- Engineering Protein Stability with Small Molecules: A Review of the ecDHFR Destabilizing Domain System.ACS chemical biology · 2026Review
- Novel cell-based assay enables FRET-based measurements of the dimerization activity of the chaperone DNAJB6.Biology methods & protocols · 2026Article
- Oxidative stress induced by sustained supraphysiological intrastriatal GDNF delivery is prevented by dose regulation.Molecular therapy. Methods & clinical development · 2023Article
- Utility of the DHFR-based destabilizing domain across mouse models of retinal degeneration and aging.iScience · 2022Article
- Regulation of protein secretion through chemical regulation of endoplasmic reticulum retention signal cleavage.Nature communications · 2022Article
- Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteinMolecular therapy. Methods & clinical development · 2021Article
- Engineered microRNA-based regulatory element permits safe high-dose miniMECP2 gene therapy in Rett mice.Brain : a journal of neurology · 2021Article
- Protocol for Designing Small-Molecule-Regulated Destabilizing Domains forSTAR protocols · 2020Article
- Protocol forSTAR protocols · 2020Article
- Simultaneous Control of Endogenous and User-Defined Genetic Pathways Using Unique ecDHFR Pharmacological Chaperones.Cell chemical biology · 2020Article
- Updates on Gene Therapy for Diabetic Retinopathy.Current diabetes reports · 2020Review
- GDNF, A Neuron-Derived Factor Upregulated in Glial Cells during Disease.Journal of clinical medicine · 2020Review
- Development and Validation of CRISPR Activator Systems for Overexpression of CB1 Receptors in Neurons.Frontiers in molecular neuroscience · 2020Article
- GDNF-mediated rescue of the nigrostriatal system depends on the degree of degeneration.Gene therapy · 2019Article
Corrections and comments
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Authors and funding
9 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Regulation of therapeutic transgene expression can increase the safety of gene therapy interventions, especially when targeting critical organs such as the brain. Although several gene expression systems have been described, none of the current systems has the required safety profile for clinical applications. Our group has previously adapted a system for novel gene regulation based on the destabilizing domain degron technology to successfully regulate glial cell-line derived neurotrophic factor in the brain (GDNF-F-DD). In the present study, we used GDNF-F-DD as a proof-of-principle molecule to fully characterize DD regulation in the brain. Our results indicate that DD could be regulated in a dose-dependent manner. In addition, GDNF-F-DD could also be induced
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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.