From gene to therapy in spinal and bulbar muscular atrophy: Are we there yet?
Abnormal polyglutamine expansions in the androgen receptor (AR) cause a muscular condition, known as Kennedy's disease or spinal and bulbar muscular atrophy (SBMA). The disease is transmitted in an X-linked fashion and is clinically characterized by weakness, atrophy and fasciculations of the l...
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| 格式: | Journal article |
| 語言: | English |
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Elsevier
2017
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