[HTML][HTML] Molecular mechanisms and therapeutics for SBMA/Kennedy's disease
FJ Arnold, DE Merry - Neurotherapeutics, 2019 - Elsevier
Spinal and bulbar muscular atrophy (SBMA) is a neuromuscular disease caused by a
polyglutamine (polyQ) expansion in the androgen receptor (AR). Despite the fact that the
monogenic cause of SBMA has been known for nearly 3 decades, there is no effective
treatment for this disease, underscoring the complexity of the pathogenic mechanisms that
lead to a loss of motor neurons and muscle in SBMA patients. In the current review, we
provide an overview of the system-wide clinical features of SBMA, summarize the structure …
polyglutamine (polyQ) expansion in the androgen receptor (AR). Despite the fact that the
monogenic cause of SBMA has been known for nearly 3 decades, there is no effective
treatment for this disease, underscoring the complexity of the pathogenic mechanisms that
lead to a loss of motor neurons and muscle in SBMA patients. In the current review, we
provide an overview of the system-wide clinical features of SBMA, summarize the structure …
