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...

Szczegółowa specyfikacja

Opis bibliograficzny
Główni autorzy: Pennuto, M, Rinaldi, C
Format: Journal article
Język:English
Wydane: Elsevier 2017