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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Những tác giả chính: Pennuto, M, Rinaldi, C
Định dạng: Journal article
Ngôn ngữ:English
Được phát hành: Elsevier 2017