CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
New England Journal of Medicine, 2021 BACKGROUND Transthyretin amyloidosis, also called ATTR amyloidosis, is a life-threatening disease characterized by progressive accumulation of misfolded transthyretin (TTR) protein in tissues, predominantly the nerves and heart.
Julian D Gillmore +2 more
exaly +2 more sources
Transthyretin Amyloidosis: Chaperone Concentration Changes and Increased Proteolysis in the Pathway to Disease. [PDF]
PLoS ONE, 2015 Transthyretin amyloidosis is a conformational pathology characterized by the extracellular formation of amyloid deposits and the progressive impairment of the peripheral nervous system.
Gonçalo da Costa +10 more
doaj +3 more sources
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
New England Journal of Medicine, 2018 BACKGROUND Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. METHODS In this phase 3 trial, we randomly assigned patients with hereditary transthyretin amyloidosis with ...
Alejandra Gonzalez-Duarte +2 more
exaly +2 more sources
Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
New England Journal of Medicine, 2018 Background Transthyretin amyloid cardiomyopathy is caused by the deposition of transthyretin amyloid fibrils in the myocardium. The deposition occurs when wild‐type or variant transthyretin becomes unstable and misfolds. Tafamidis binds to transthyretin,
Mathew S Maurer +2 more
exaly +2 more sources
Structure of human transthyretin complexed with bromophenols: a new mode of binding [PDF]
, 2000 The binding of two organohalogen substances, pentabromophenol (PBP) and 2,4,6-tribromophenol (TBP), to human transthyretin (TTR), a thyroid hormone transport protein, has been studied by in vitro competitive binding assays and by X-ray crystallography ...
Bergman, A +5 more
core +4 more sources
Natural History, Quality of Life, and Outcome in Cardiac Transthyretin Amyloidosis
Circulation, 2019 BACKGROUND Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is an increasingly recognized cause of heart failure in older individuals. We sought to characterize the natural history of ATTR-CM and compare outcomes and quality of life among patients with
Marianna Fontana +2 more
exaly +2 more sources
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
New England Journal of Medicine, 2018 BACKGROUND Hereditary transthyretin amyloidosis is caused by pathogenic single‐nucleotide variants in the gene encoding transthyretin (TTR) that induce transthyretin misfolding and systemic deposition of amyloid. Progressive amyloid accumulation leads to
John L Berk +2 more
exaly +2 more sources
Systemic Transthyretin Amyloidosis Incidentally Diagnosed With Prostate Biopsy: A Case Report [PDF]
IJU Case ReportsIntroduction The diagnosis of systemic transthyretin amyloidosis based on prostate biopsy is very rare, and we report this case with a literature review.
Michihide Nakamura +14 more
doaj +2 more sources
A new staging system for cardiac transthyretin amyloidosis
European Heart Journal, 2018 Julian D Gillmore +2 more
exaly +2 more sources
Patisiran Treatment in Patients with Transthyretin Cardiac Amyloidosis.
New England Journal of Medicine, 2023 BACKGROUND Transthyretin amyloidosis, also called ATTR amyloidosis, is associated with accumulation of ATTR amyloid deposits in the heart and commonly manifests as progressive cardiomyopathy. Patisiran, an RNA interference therapeutic agent, inhibits the
M. S. Maurer +36 more
semanticscholar +1 more source