Article
Selective modulation of the androgen receptor AF2 domain rescues degeneration in spinal bulbar muscular atrophy.
Nature medicine - 1 May 2018
Badders Nisha M, Korff Ane, Miranda Helen C, Vuppala Pradeep K, Smith Rebecca B, Winborn Brett J, Quemin Emmanuelle R, Sopher Bryce L, Dearman Jennifer, Messing James, Kim Nam Chul, Moore Jennifer, Freibaum Brian D, Kanagaraj Anderson P, Fan Baochang, Tillman Heather, Chen Ping-Chung, Wang Yingzhe, Freeman Burgess B, Li Yimei, Kim Hong Joo, La Spada Albert R, Taylor J Paul
Abstract excerpt
Spinal bulbar muscular atrophy (SBMA) is a motor neuron disease caused by toxic gain of function of the androgen receptor (AR). Previously, we found that co-regulator binding through the activation function-2 (AF2) domain of AR is essential for pathogenesis, suggesting that AF2 may be a potential drug target for selective modulation of toxic AR activity. We screened previously identified AF2 modulators for their...
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