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Current Status of Treatment of Spinal and Bulbar Muscular Atrophy
Joint Authors
Sobue, Gen
Tanaka, Fumiaki
Katsuno, Masahisa
Banno, Haruhiko
Suzuki, Keisuke
Adachi, Hiroaki
Source
Issue
Vol. 2012, Issue 2012 (31 Dec. 2012), pp.1-8, 8 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2012-06-07
Country of Publication
Egypt
No. of Pages
8
Main Subjects
Abstract EN
Spinal and bulbar muscular atrophy (SBMA) is the first member identified among polyglutamine diseases characterized by slowly progressive muscle weakness and atrophy of the bulbar, facial, and limb muscles pathologically associated with motor neuron loss in the spinal cord and brainstem.
Androgen receptor (AR), a disease-causing protein of SBMA, is a well-characterized ligand-activated transcription factor, and androgen binding induces nuclear translocation, conformational change and recruitment of coregulators for transactivation of AR target genes.
Some therapeutic strategies for SBMA are based on these native functions of AR.
Since ligand-induced nuclear translocation of mutant AR has been shown to be a critical step in motor neuron degeneration in SBMA, androgen deprivation therapies using leuprorelin and dutasteride have been developed and translated into clinical trials.
Although the results of these trials are inconclusive, renewed clinical trials with more sophisticated design might prove the effectiveness of hormonal intervention in the near future.
Furthermore, based on the normal function of AR, therapies targeted for conformational changes of AR including amino-terminal (N) and carboxy-terminal (C) (N/C) interaction and transcriptional coregulators might be promising.
Other treatments targeted for mitochondrial function, ubiquitin-proteasome system (UPS), and autophagy could be applicable for all types of polyglutamine diseases.
American Psychological Association (APA)
Tanaka, Fumiaki& Katsuno, Masahisa& Banno, Haruhiko& Suzuki, Keisuke& Adachi, Hiroaki& Sobue, Gen. 2012. Current Status of Treatment of Spinal and Bulbar Muscular Atrophy. Neural Plasticity،Vol. 2012, no. 2012, pp.1-8.
https://search.emarefa.net/detail/BIM-1002406
Modern Language Association (MLA)
Tanaka, Fumiaki…[et al.]. Current Status of Treatment of Spinal and Bulbar Muscular Atrophy. Neural Plasticity No. 2012 (2012), pp.1-8.
https://search.emarefa.net/detail/BIM-1002406
American Medical Association (AMA)
Tanaka, Fumiaki& Katsuno, Masahisa& Banno, Haruhiko& Suzuki, Keisuke& Adachi, Hiroaki& Sobue, Gen. Current Status of Treatment of Spinal and Bulbar Muscular Atrophy. Neural Plasticity. 2012. Vol. 2012, no. 2012, pp.1-8.
https://search.emarefa.net/detail/BIM-1002406
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1002406