ReviewMolecular therapy. Nucleic acids2025
Realizing the therapeutic potential of rapid knockdown of transthyretin via RNA interference in transthyretin amyloidosis.
Review in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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
1 citing paper in PubMed.
- Gene therapy for liver diseases: methods, challenges and opportunities.Journal of nanobiotechnology · 2026Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
RNA interference (RNAi) is a natural biological mechanism that regulates gene expression by degrading a specific target messenger RNA, resulting in reduced production of the target protein. RNAi therapeutics can silence the production of a disease-causing protein in a specific organ or cell type and have the advantage of having a long duration of effect due to their inherently stable duplex structure and chemical modifications. Following the development of RNAi-based platform technologies, a growing number of RNAi therapeutics have now been approved across a range of diseases. Transthyretin amyloidosis (ATTR) is a progressive, debilitating, and fatal disease caused by ongoing accumulation of toxic transthyretin (TTR) amyloid deposits in multiple organs and tissues. This review describes the clinical development of patisiran and vutrisiran, which target the hepatic production of TTR for the treatment of ATTR amyloidosis. Findings from clinical studies show that rapid knockdown of TTR by RNAi therapeutics initiates a cascade of effects that ultimately leads to benefits across multiple clinical, quality of life, imaging, and biomarker disease measures of ATTR amyloidosis disease progression.
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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.