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Myasthenia Gravis

Efficacious mRNA-targeted therapies hold some advantages

Published in Gene Therapy Weekly, May 1st, 2003

Researchers in Israel report efficacious treatment with minute doses of an antisense oligonucleotide of a single acetylcholinesterase splicing variant in rats with experimental autoimmune myasthenia gravis.

"Alternative splicing induces, under abnormal cholinergic neurotransmission, overproduction of the rare 'readthrough' acetylcholinesterase (AChE) variant AChE-R. We explored the pathophysiological relevance of this phenomenon in patients with myasthenia gravis (MG) and rats with experimental autoimmune MG (EAMG), neuromuscular junction diseases with depleted acetylcholine receptors," said T. Brenner and colleagues, Hebrew University of Jerusalem.

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