Journal of cardiovascular pharmacology, 2021-11, Vol.78 (5), p.e641-e647
2021
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Details
- Autor(en) / Beteiligte
- Titel
- Disease-Modifying Treatments for Transthyretin Amyloidosis
- Ist Teil von
- Journal of cardiovascular pharmacology, 2021-11, Vol.78 (5), p.e641-e647
- Ort / Verlag
- United States
- Erscheinungsjahr
- 2021
- Quelle
- MEDLINE
- Beschreibungen/Notizen
- The transthyretin (TTR) amyloidoses result from misfolding of the protein leading to fibril formation and aggregation as amyloid deposits in predominantly the cardiovascular and nervous systems. Cardiac involvement can manifest as heart failure, arrhythmias, and valvular disease. Neurologic involvement can cause sensorimotor polyneuropathies, mononeuropathies, and dysautonomia. Previously, treatment has focused on management of these symptoms and disease sequelae, with a high rate of mortality due to the absence of disease-modifying therapies. In this article, we review novel treatments focusing on 3 mechanistic pathways: (1) silencing of the TTR gene to suppress production, (2) stabilizing of TTR tetramers to prevent misfolding, or (3) disrupting of existing TTR amyloid fibrils to promote reabsorption.
- Sprache
- Englisch
- Identifikatoren
- ISSN: 0160-2446eISSN: 1533-4023DOI: 10.1097/FJC.0000000000001115Titel-ID: cdi_pubmed_primary_34321398
- Format
- –
- Schlagworte
- Amyloid - drug effects, Amyloid - metabolism, Amyloid Neuropathies, Familial - genetics, Amyloid Neuropathies, Familial - metabolism, Amyloid Neuropathies, Familial - pathology, Amyloid Neuropathies, Familial - therapy, Animals, Cardiomyopathies - genetics, Cardiomyopathies - metabolism, Cardiomyopathies - pathology, Cardiomyopathies - therapy, Cardiovascular Agents - adverse effects, Cardiovascular Agents - therapeutic use, Gene Silencing, Genetic Predisposition to Disease, Genetic Therapy, Humans, Mutation, Myocytes, Cardiac - drug effects, Myocytes, Cardiac - metabolism, Myocytes, Cardiac - pathology, Phenotype, Prealbumin - genetics, Prealbumin - metabolism, Protein Stability