ArticleGene therapy2011
Preclinical development of siRNA therapeutics for AL amyloidosis.
Article in Gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 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
16 citing papers in PubMed, 38 citations in OpenAlex.
- In vitro and in vivo disease models of cardiac amyloidosis: progress, pitfalls, and potential.Cardiovascular research · 2025Review
- Bad players in AL amyloidosis in the current era of treatment.Expert review of hematology · 2023Article
- Systemic amyloidosis: moving into the spotlight.Leukemia · 2020Review
- Hematological Malignancies and Arterial Thromboembolism.Indian journal of hematology & blood transfusion : an official journal of Indian Society of Hematology and Blood Transfusion · 2019Review
- A novel xenograft mouse model for testing approaches targeting human kappa light-chain diseases.Gene therapy · 2019Article
- Animal models of monoclonal immunoglobulin-related renal diseases.Nature reviews. Nephrology · 2018Review
- siRNA targeting the κ light chain constant region: preclinical testing of an approach to nonfibrillar and fibrillar light chain deposition diseases.Gene therapy · 2016Article
- Viewing Extrinsic Proteotoxic Stress Through the Lens of Amyloid Cardiomyopathy.Physiology (Bethesda, Md.) · 2016Review
- The Prevalence and Management of Systemic Amyloidosis in Western Countries.Kidney diseases (Basel, Switzerland) · 2016Review
- Targeting protein aggregation for the treatment of degenerative diseases.Nature reviews. Drug discovery · 2015Review
- Update on treatment of light chain amyloidosis.Haematologica · 2014Review
- Light chain amyloidosis: the heart of the problem.Haematologica · 2013Article
- Plasmocytoma-induced intertriginous amyloid purpura.Annals of dermatology · 2013Article
- Current perspectives on cardiac amyloidosis.American journal of physiology. Heart and circulatory physiology · 2012Review
- Amyloid formation in light chain amyloidosis.Current topics in medicinal chemistry · 2012Review
- Article
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 2 institutions in 1 country.
Funding
Abstract
Amyloid light chain (AL) amyloidosis is a rare hematologic disorder characterized by the accumulation of a misfolded monoclonal immunoglobulin (Ig) light chain (LC) as fibrillar protein deposits. Current treatments, including cytotoxic chemotherapy and immunomodulatory therapy, are directed at killing the plasma cells that produce the LCs, but have significant toxicity for other cell types. We have designed small interfering RNAs (siRNAs) targeting the amyloidogenic LC messenger RNA (mRNA) in order to reduce expression of the amyloid precursor protein. Using nanomolar concentrations of siRNAs, we have inhibited synthesis of LC in transfected cells in vitro in a dose-dependent fashion. Furthermore, in an in vivo plasmacytoma mouse model of AL amyloidosis, we have demonstrated that these siRNAs can significantly reduce local production and circulating levels of LC. This model system highlights the therapeutic potential of siRNA for AL amyloidosis.
Indexed as
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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.