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Overexpression of IGF-1 in muscle attenuates disease in a mouse model of spinal and bulbar muscular atrophy.

Title: Overexpression of IGF-1 in muscle attenuates disease in a mouse model of spinal and bulbar muscular atrophy.
Authors: Palazzolo, Isabella; Stack, Conor; Kong, Lingling; Musaro, Antonio; Adachi, Hiroaki; Katsuno, Masahisa; Sobue, Gen; Taylor, Jean Paul; Sumner, Charlotte J.; Fischbeck, Kenneth H.; Pennuto, Maria
Contributors: Palazzolo, Isabella; Stack, Conor; Kong, Lingling; Musaro, Antonio; Adachi, Hiroaki; Katsuno, Masahisa; Sobue, Gen; Taylor, Jean Paul; Sumner, Charlotte J.; Fischbeck, Kenneth H.; Pennuto, Maria
Publisher Information: USA
Publication Year: 2009
Collection: Università degli Studi di Trento: CINECA IRIS
Description: Expansion of a polyglutamine tract in the androgen receptor (AR) causes spinal and bulbar muscular atrophy (SBMA). We previously showed that Akt-mediated phosphorylation of AR reduces ligand binding and attenuates the mutant AR toxicity. Here, we show that in culture insulin-like growth factor 1 (IGF-1) reduces AR aggregation and increases AR clearance via the ubiquitin-proteasome system through phosphorylation of AR by Akt. In vivo, SBMA transgenic mice overexpressing a muscle-specific isoform of IGF-1 selectively in skeletal muscle show evidence of increased Akt activation and AR phosphorylation and decreased AR aggregation. Augmentation of IGF-1/Akt signaling rescues behavioral and histopathological abnormalities, extends the life span, and reduces both muscle and spinal cord pathology of SBMA mice. This study establishes IGF-1/Akt-mediated inactivation of mutant AR as a strategy to counteract disease in vivo and demonstrates that skeletal muscle is a viable target tissue for therapeutic intervention in SBMA.
Document Type: article in journal/newspaper
File Description: STAMPA
Language: English
Relation: info:eu-repo/semantics/altIdentifier/pmid/19679072; info:eu-repo/semantics/altIdentifier/wos/WOS:000269285500007; volume:2009, 63; issue:3; firstpage:316; lastpage:328; numberofpages:13; journal:NEURON; https://hdl.handle.net/11572/98247
DOI: 10.1016/j.neuron.2009.07.019
Availability: https://hdl.handle.net/11572/98247; https://doi.org/10.1016/j.neuron.2009.07.019; https://www.cell.com/neuron/fulltext/S0896-6273(09)00548-0
Rights: info:eu-repo/semantics/openAccess ; license:Tutti i diritti riservati (All rights reserved)
Accession Number: edsbas.107DFD9A
Database: BASE