ArticleiScience2025
A luminescence-based biosensor to measure endogenous UBE3A activity.
Article in iScience, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
What it found
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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.
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
2 citing papers in PubMed.
- Review
- A dual-reporter mouse for therapeutic discovery in Angelman syndrome.JCI insight · 2026Article
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Authors and funding
6 authors.
Funding
Abstract
Loss- and gain-of-function (LOF and GOF) mutations in the E3 ubiquitin ligase UBE3A cause distinct neurodevelopmental disorders. The AZUL domain of UBE3A binds with low nanomolar affinity to a 45 amino acid (aa) region of PSMD4. We fused this 45-aa sequence to Firefly luciferase to generate a luminescence-based biosensor, Firefly-45aa, that acts as an artificial UBE3A substrate and exhibits exceptional sensitivity in measuring UBE3A activity. Testing UBE3A variants using Firefly-45aa in HEK293T cells revealed distinct biosensor activity profiles, enabling the classification of LOF and GOF mutations based on maximum activity, inhibitory concentration-50, and activity per unit of protein. Some strong LOF mutations had dominant negative activity. In addition, Firefly-45aa can be used to quantify endogenous UBE3A activity in primary cells from Angelman syndrome and GOF mouse models. This biosensor reveals a mutational spectrum of UBE3A enzyme activity and is a sensitive tool for functional characterization and therapeutic development in UBE3A-related disorders.
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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.