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Article

Synergic prodegradative activity of Bicalutamide and trehalose on the mutant androgen receptor responsible for spinal and bulbar muscular atrophy.

Human molecular genetics - 1 Jan 2015

Giorgetti Elisa, Rusmini Paola, Crippa Valeria, Cristofani Riccardo, Boncoraglio Alessandra, Cicardi Maria E, Galbiati Mariarita, Poletti Angelo

Abstract excerpt

Spinal and bulbar muscular atrophy (SBMA) is an X-linked motoneuron disease due to a CAG triplet-repeat expansion in the androgen receptor (AR) gene, which is translated into an elongated polyglutamine (polyQ) tract in AR protein (ARpolyQ). ARpolyQ toxicity is activated by the AR ligand testosterone (or dihydrotestosterone), and the polyQ triggers ARpolyQ misfolding and aggregation in spinal cord motoneurons and...

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Identifiers

PMID: 25122660
DOI: 10.1093/hmg/ddu419
PMCID: PMC4262493

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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