ArticleAmyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis2016
Novel conformation-specific monoclonal antibodies against amyloidogenic forms of transthyretin.
Article in Amyloid : the international journal of experimental and clinical investigation : the official journal of the International Society of Amyloidosis, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 45 papers.
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Who cites it
45 citing papers in PubMed, 86 citations in OpenAlex.
- Trial
- Biologics for cardiovascular diseases: from bench to bedside.Signal transduction and targeted therapy · 2026Review
- Emerging Pharmacological Strategies for Cardiac Amyloidosis: A Qualitative Analysis of Interventional Clinical Trials Registered on ClinicalTrials.Gov.Journal of clinical medicine · 2026Review
- A functional role for spontaneously occurring natural anti-transthyretin antibodies from patients with transthyretin cardiac amyloidosis.European journal of heart failure · 2025Article
- Transthyretin Amyloid Cardiomyopathy Treatment: An Updated Review.Journal of clinical medicine · 2025Review
- Transthyretin Amyloid Cardiomyopathy-2025 Update: Current Diagnostic Approaches and Emerging Therapeutic Options.Journal of clinical medicine · 2025Review
- Transthyretin Amyloid Cardiomyopathy: The Plot Thickens as Novel Therapies Emerge.US cardiology · 2025Review
- Structural Basis for Monoclonal Antibody Therapy for Transthyretin Amyloidosis.Pharmaceuticals (Basel, Switzerland) · 2024Review
- Neutrophils enhance the clearance of systemic amyloid deposits in a murine amyloidoma model.Frontiers in immunology · 2024Article
- Macrophage Phagocytes Amyloid Fibrils in Giant Cell Myocarditis.Internal medicine (Tokyo, Japan) · 2023Article
- Emerging Therapies for Transthyretin Amyloidosis.Current oncology reports · 2023Review
- Natural history and survival rate of familial amyloidosis with polyneuropathy: A nationwide databank.Annals of clinical and translational neurology · 2023Article
- Transthyretin Stabilizers and Seeding Inhibitors as Therapies for Amyloid Transthyretin Cardiomyopathy.Pharmaceutics · 2023Review
- Development and characterization of a prototypic pan-amyloid clearing agent - a novel murine peptide-immunoglobulin fusion.Frontiers in immunology · 2023Article
- Leptomeningeal Disease Secondary to Thr60Ala Transthyretin Amyloidosis: Case Report and Review of the Literature.The Neurohospitalist · 2023Article
- Human Islet Amyloid Polypeptide (hIAPP) Protofibril-Specific Antibodies for Detection and Treatment of Type 2 Diabetes.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2022Article
- Article
- Transthyretin proteoforms of intraocular origin in human subretinal fluid.Experimental eye research · 2022Article
- Targeted treatments of AL and ATTR amyloidosis.Heart failure reviews · 2022Review
- Targeting Amyloid Fibrils by Passive Immunotherapy in Systemic Amyloidosis.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2022Review
Corrections and comments
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Authors and funding
14 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
introductionTransthyretin amyloidosis (ATTR amyloidosis) is caused by the misfolding and deposition of the transthyretin (TTR) protein and results in progressive multi-organ dysfunction. TTR epitopes exposed by dissociation and misfolding are targets for immunotherapeutic antibodies. We developed and characterized antibodies that selectively bound to misfolded, non-native conformations of TTR.
methodsAntibody clones were generated by immunizing mice with an antigenic peptide comprising a cryptotope within the TTR sequence and screened for specific binding to non-native TTR conformations, suppression of in vitro TTR fibrillogenesis, promotion of antibody-dependent phagocytic uptake of mis-folded TTR and specific immunolabeling of ATTR amyloidosis patient-derived tissue.
resultsFour identified monoclonal antibodies were characterized. These antibodies selectively bound the target epitope on monomeric and non-native misfolded forms of TTR and strongly suppressed TTR fibril formation in vitro. These antibodies bound fluorescently tagged aggregated TTR, targeting it for phagocytic uptake by macrophage THP-1 cells, and amyloid-positive TTR deposits in heart tissue from patients with ATTR amyloidosis, but did not bind to other types of amyloid deposits or normal tissue.
conclusionsConformation-specific anti-TTR antibodies selectively bind amyloidogenic but not native TTR. These novel antibodies may be therapeutically useful in preventing deposition and promoting clearance of TTR amyloid and in diagnosing TTR amyloidosis.
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