C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/7088 (2006.01) A61P 21/04 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2458806
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.
L'invention concerne un oligonucléotide antisens ciblant la zone de codage de l'acétylcholinestérase humaine (AChE), qui supprime de manière sélective l'isoforme AchE-R de l'enzyme. L'oligonucléotide antisens peut être utilisé dans le traitement et/ou la prévention de troubles neuromusculaires, de préférence myasthenia gravis. En outre, il peut pénétrer la barrière hémato-encéphalique et détruire l'AchE-R à l'intérieur des neurones du système nerveux central, tout en servant de porteur permettant de transporter des molécules à travers ladite barrière hémato-encéphalique.
Goudreau Gage Dubuc
Yissum Research Development Company Of The Hebrew University In
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