Treating Protein Misfolding Diseases: Therapeutic Successes Against Systemic Amyloidoses.

ABSTRACT: Misfolding and extracellular deposition of proteins is the hallmark of a heterogeneous group of conditions collectively termed protein misfolding and deposition diseases or amyloidoses. These include both localized (e.g. Alzheimer's disease, prion diseases, type 2 diabetes mellitus) and systemic amyloidoses. Historically regarded as a group of maladies with limited, even inexistent, therapeutic options, some forms of systemic amyloidoses have recently witnessed a series of unparalleled therapeutic successes, positively impacting on their natural history and sometimes even on their incidence. In this review article we will revisit the most relevant of these accomplishments. Collectively, current evidence converges towards a crucial role of an early and conspicuous reduction or stabilization

SUBMITTER: Nevone A 

PROVIDER: S-EPMC7366848 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature