Indeed, antisense oligonucleotides specifically suppressing peripheral, but not central nervous system (CNS), AR gene expression rescued muscle deficits extending lifespan of a male mouse (knock-in) model of SBMA (11); muscle-specific excision of human AR121Q present in a bacterial artificial chromosome (BAC) vectors transgenic mouse model of SBMA resulted in a full rescue from the typical aberrant phenotype, including prevention of weight loss, motor phenotypes, muscle pathology and motoneuropathy. This evidence concerns the gene AR and Kennedy disease.