Ontology highlight
ABSTRACT:
SUBMITTER: Pourshafie N
PROVIDER: S-EPMC4881766 | biostudies-literature | 2016 May
REPOSITORIES: biostudies-literature
Pourshafie Naemeh N Lee Philip R PR Chen Ke-Lian KL Harmison George G GG Bott Laura C LC Katsuno Masahisa M Sobue Gen G Burnett Barrington G BG Fischbeck Kenneth H KH Rinaldi Carlo C
Molecular therapy : the journal of the American Society of Gene Therapy 20160112 5
Spinal and bulbar muscular atrophy (SBMA) is a currently untreatable adult-onset neuromuscular disease caused by expansion of a polyglutamine repeat in the androgen receptor (AR). In SBMA, as in other polyglutamine diseases, a toxic gain of function in the mutant protein is an important factor in the disease mechanism; therefore, reducing the mutant protein holds promise as an effective treatment strategy. In this work, we evaluated a microRNA (miRNA) to reduce AR expression. From a list of pred ...[more]