Antisense oligonucleotide against human acetylcholinesterase (AChE) and uses thereof
AssigneeYissum Research Development Company of the Hebrew University of Jerusalem
InventorsHermona Soreq, Tama Evron
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Abstract
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.
Claims9
A method of treating a progressive neuromuscular disorder, for improving stamina, for treating chronic muscle fatigue, or for treating myasthemia gravis, or a disorder associated with excess of AChE mRNA or protein, which comprises administering a composition comprising an antisense oligonucleotide hEN101, defined by SEQ ID NO:1, to a patient in need thereof.
Dependencies are inferred from the claim text.