Antisense oligonucleotide against human acetylcholinesterase (AChE) and uses thereof

US7074915B2 · kind B2 · utility

Assignee

• YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD. · IL

Inventors

• Hermona Soreq · Nesher, IL
• Tama Evron · Mevaseret Tsiyon, IL
• Shlomo Saidman · Rishon LeZion, IL

Key dates

• Filing date: Mar 27, 2003
• Grant date: Jul 11, 2006
• Expiry date: Sep 30, 2023

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/53
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.