ASC-J9 ameliorates spinal and bulbar muscular atrophy phenotype via degradation of androgen receptor

Z Yang, YJ Chang, IC Yu, S Yeh, CC Wu, H Miyamoto… - Nature medicine, 2007 - nature.com
Z Yang, YJ Chang, IC Yu, S Yeh, CC Wu, H Miyamoto, DE Merry, G Sobue, LM Chen…
Nature medicine, 2007nature.com
Motor neuron degeneration resulting from the aggregation of the androgen receptor with an
expanded polyglutamine tract (AR-polyQ) has been linked to the development of spinal and
bulbar muscular atrophy (SBMA or Kennedy disease). Here we report that adding 5-hydroxy-
1, 7-bis (3, 4-dimethoxyphenyl)-1, 4, 6-heptatrien-3-one (ASC-J9) disrupts the interaction
between AR and its coregulators, and also increases cell survival by decreasing AR-polyQ
nuclear aggregation and increasing AR-polyQ degradation in cultured cells. Intraperitoneal …
Abstract
Motor neuron degeneration resulting from the aggregation of the androgen receptor with an expanded polyglutamine tract (AR-polyQ) has been linked to the development of spinal and bulbar muscular atrophy (SBMA or Kennedy disease). Here we report that adding 5-hydroxy-1,7-bis(3,4-dimethoxyphenyl)-1,4,6-heptatrien-3-one (ASC-J9) disrupts the interaction between AR and its coregulators, and also increases cell survival by decreasing AR-polyQ nuclear aggregation and increasing AR-polyQ degradation in cultured cells. Intraperitoneal injection of ASC-J9 into AR-polyQ transgenic SBMA mice markedly improved disease symptoms, as seen by a reduction in muscular atrophy. Notably, unlike previous approaches in which surgical or chemical castration was used to reduce SBMA symptoms, ASC-J9 treatment ameliorated SBMA symptoms by decreasing AR-97Q aggregation and increasing VEGF164 expression with little change of serum testosterone. Moreover, mice treated with ASC-J9 retained normal sexual function and fertility. Collectively, our results point to a better therapeutic and preventative approach to treating SBMA, by disrupting the interaction between AR and AR coregulators.
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