Spinal and bulbar muscular atrophy (SBMA) is caused by the expansion of a polyglutamine-encoding stretch in the androgen receptor gene, and is characterized by the presence of androgen-receptor-containing nuclear inclusions in brainstem and spinal motor neurons. As is the case for every polyglutamine disease, there is no effective treatment for SBMA. However, this situation might change soon, following the report by Katsuno et al. in Nature Medicine that hormonal therapy with leuprorelin abrogates SBMA phenotypes in a mouse model of the disease.
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