Target intelligence / Profile preview

Amyloid fibril (tissue deposits) (None)

Target
None
Molecular classification
Other (aggregated protein material)
01

Overview

Amyloid deposits are insoluble fibrillar aggregates of misfolded proteins that accumulate in tissues and organs, disrupting normal structure and function. They are characteristic of a group of diseases called amyloidoses, which can be systemic or localized. Each amyloidosis subtype is defined by the precursor protein forming the fibrils: common variants include immunoglobulin light chains (AL amyloidosis), serum amyloid A (AA amyloidosis), transthyretin (ATTR, in familial or senile amyloidosis), and amyloid-beta (Aβ, in Alzheimer's disease). Diagnosis relies on tissue biopsy and specialized staining (Congo red), while therapy aims to reduce precursor protein production and clear deposits. Amyloid accumulation is linked to severe, often multisystem disease, and disease management involves both direct and indirect targeting of underlying protein misfolding and deposition.

Other names
Amyloid depositsAmyloid accumulationAmyloid fibril depositsAmyloid plaque
02

Mechanism of action

Inhibit precursor protein production (e.g., stabilize or silence genes); Facilitate clearance or reduce aggregation of amyloid fibrils; Immunotherapy (experimental: antibodies to remove deposits).

03

Biological functions

Other (pathological aggregates interfering with tissue and organ function)
04

Disease associations

Neurodegenerative diseaseCardiovascular diseaseRenal diseaseChronic inflammatory diseaseCancer
05

Safety considerations

Organ failure due to extensive depositsToxicity of chemotherapy agents used to treat precursor diseasesAmyloid extraction therapies can trigger inflammatory responsesUnderdiagnosis due to nonspecific symptoms
06

Interacting drugs

Tafamidis

8 more in the full profile.

07

Biomarkers

Congo red staining of biopsiesSerum immunoglobulin free light chainsSerum amyloid A proteinTransthyretin variantsCardiac biomarkers: NT-proBNP, troponin

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