Neuropathology and Therapeutic Intervention in Spinal and Bulbar Muscular Atrophy

International Journal of Molecular Sciences

Haruhiko Banno¹  Masahisa Katsuno¹  Keisuke Suzuki¹  Fumiaki Tanaka¹ 

[1] Department of Neurology, Nagoya University Graduate School of Medicine / 65 Tsurumai-cho, Showa-ku, Nagoya 466-8550, Japan; E-Mails:

关键词: Spinal and bulbar muscular atrophy (SBMA);    polyglutamine;    androgen receptor (AR);    leuprorelin acetate;   

DOI  :  10.3390/ijms10031000

来源: mdpi

【 摘 要 】

Spinal and bulbar muscular atrophy (SBMA) is a hereditary motor neuron disease caused by the expansion of a polyglutamine tract in the androgen receptor (AR). The histopathological finding in SBMA is loss of lower motor neurons in the anterior horn of the spinal cord as well as in the brainstem motor nuclei. Animal studies have revealed that the pathogenesis of SBMA depends on the level of serum testosterone, and that androgen deprivation mitigates neurodegeneration through inhibition of nuclear accumulation of the pathogenic AR. Heat shock proteins, ubiquitin-proteasome system and transcriptional regulation are also potential targets of therapy development for SBMA.

【 授权许可】

CC BY   
© 2009 by the authors; licensee Molecular Diversity Preservation International, Basel, Switzerland. This article is an open-access article distributed under the terms and conditions of the Creative Commons Attribution license (http://creativecommons.org/licenses/by/3.0/).