MUSCULAR DYSTROPHY THERAPIES - AN OVERVIEW

Recently, the antibiotic gentamicin is examined in boys with DMD, about ten% of whom have inherited a dystrophin gene using a premature end codon that halts the synthesis in the protein N-terminal on the dystroglycan binding region. Gentamicin suppresses chain termination, permitting some full-duration dystrophin to become made. This ends in enhanc