New treatments for myasthenia: a focus on antisense oligonucleotides.
Autoimmune myasthenia gravis (MG) is a neuromuscular disorder caused by autoantibodies directed against the acetylcholine receptor (AChR). Current symptomatic therapy is based on acetylcholinesterase (AChE) drugs. The available long-term current therapy includes steroids and other immunomodulatory agents. MG is associated with the production of a soluble, rare isoform of AChE, also referred as the "read-through" transcript (AChE-R). Monarsen (EN101) is a synthetic antisense compound directed against the AChE gene. Monarsen was administered in 16 patients with MG and 14 patients achieved a clinically significant response. The drug is now in a Phase II study. Further investigations are required to confirm its long-term effects.
IRCCS S Camillo, Via Alberoni, Venice, Italy. corrado.angelini@unipd.it
Corrado
C
Angelini
Sara
S
Martignago
Michela
M
Bisciglia
D000110:Acetylcholinesterase / Q000235:genetics / Q000378:metabolism; D000818:Animals; D002800:Cholinesterase Inhibitors / Q000494:pharmacology / Q000627:therapeutic use; D006801:Humans; D009157:Myasthenia Gravis / Q000276:immunology / Q000503:physiopathology / Q000628:therapy*; D009838:Oligodeoxyribonucleotides / Q000627:therapeutic use*; D016376:Oligonucleotides, Antisense / Q000494:pharmacology / Q000627:therapeutic use*; D020033:Protein Isoforms; D011950:Receptors, Cholinergic / Q000276:immunology
D002800:Cholinesterase Inhibitors; C477243:EN101; D009838:Oligodeoxyribonucleotides; D016376:Oligonucleotides, Antisense; D020033:Protein Isoforms; D011950:Receptors, Cholinergic; D000110:Acetylcholinesterase
EN101; acetyl cholinesterase; antisense oligonucleotides; myasthenia gravis