ArticleMolecular therapy : the journal of the American Society of Gene Therapy2018
A Drug-Tunable Gene Therapy for Broad-Spectrum Protection against Retinal Degeneration.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
The trial behind it
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
17 citing papers in PubMed, 32 citations in OpenAlex.
- Identification of Recombinant Adeno-Associated Virus Serotypes by Matrix-Assisted Laser Desorption/Ionization Mass Spectrometry.Analytical chemistry · 2026Article
- Time-Resolved Oxidative Stress and Apoptosis in Murine Retina under Acute Hypobaric Hypoxia with Parallel Activation of the LIF-JAK-STAT3 Axis.Molecular neurobiology · 2026Article
- A programmable genetic platform for engineering noninvasive biosensors.Science advances · 2026Article
- A programmable genetic platform for engineering noninvasive biosensors.bioRxiv : the preprint server for biology · 2025Article
- Modulation of retinal inflammation delays degeneration in a mouse model of geographic atrophy.Scientific reports · 2025Article
- Riboswitch-controlled IL-12 gene therapy reduces hepatocellular cancer in mice.Frontiers in immunology · 2024Article
- Intravitreal Injection of ZYAN1 Restored Autophagy and Alleviated Oxidative Stress in Degenerating Retina via the HIF-1α/BNIP3 Pathway.Antioxidants (Basel, Switzerland) · 2023Article
- Cell-specific regulation of gene expression using splicing-dependent frameshifting.Nature communications · 2022Article
- Inclusion of a degron reduces levelsof undesired inteins after AAV-mediated proteinMolecular therapy. Methods & clinical development · 2021Article
- Retinal Gene Therapy for Usher Syndrome: Current Developments, Challenges, and Perspectives.International ophthalmology clinics · 2021Article
- Cell Replacement Therapy for Retinal and Optic Nerve Diseases: Cell Sources, Clinical Trials and Challenges.Pharmaceutics · 2021Review
- Identified GNGT1 and NMU as Combined Diagnosis Biomarker of Non-Small-Cell Lung Cancer Utilizing Bioinformatics and Logistic Regression.Disease markers · 2021Article
- Paving the way for small-molecule drug discovery.American journal of translational research · 2021Review
- Clarin-1 expression in adult mouse and human retina highlights a role of Müller glia in Usher syndrome.The Journal of pathology · 2020Article
- Non-antibiotic Small-Molecule Regulation of DHFR-Based Destabilizing DomainsMolecular therapy. Methods & clinical development · 2019Article
- AKT3 Gene Transfer Promotes Anabolic Reprogramming and Photoreceptor Neuroprotection in a Pre-clinical Model of Retinitis Pigmentosa.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- New Therapeutic Options for Advanced Hepatocellular Carcinoma.Cancer control : journal of the Moffitt Cancer CenterArticle
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Retinal degenerations are a large cluster of diseases characterized by the irreversible loss of light-sensitive photoreceptors that impairs the vision of 9.1 million people in the US. An attractive treatment option is to use gene therapy to deliver broad-spectrum neuroprotective factors. However, this approach has had limited clinical translation because of the inability to control transgene expression. To address this problem, we generated an adeno-associated virus vector named RPF2 that was engineered to express domains of leukemia inhibitory factor fused to the destabilization domain of bacterial dihydrofolate reductase. Fusion proteins containing the destabilization domain are degraded in mammalian cells but can be stabilized with the binding of the drug trimethoprim. Our data show that expression levels of RPF2 are tightly regulated by the dose of trimethoprim and can be reversed by trimethoprim withdrawal. We further show that stabilized RPF2 can protect photoreceptors and prevent blindness in treated mice.
Indexed as
Identifiers
What OpenQuestion holds
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.