ArticleMolecular therapy. Nucleic acids2026
Targeted gene editing of
Article in Molecular therapy. Nucleic acids, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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.
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0 citing papers in PubMed.
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Authors and funding
4 authors.
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Abstract
Deep-intronic variants activating pseudoexons (PEs) are a common cause for monogenic diseases. Removal of the PE region predictably corrects the splicing defect, offering a potential therapeutic strategy. Previous studies, including our own, have identified an 84-bp PE in intron 14 of the gene
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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.