The invention relates to an antisense oligonucleotide targeted to the
coding region of the human acetylcholinesterase (AChE), which selectively
suppresses the AChE-R isoform of the enzyme. The antisense
oligonucleotide is intended for use in the treatment and/or prevention of
neuromuscular disorders, preferably myasthenia gravis. In addition, it
can penetrate the blood-brain barrier (BBB) and destroy AChE-R within
central nervous system neurons, while also serving as a carrier to
transport molecules across the BBB.