YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREWUNIVERSITY OF JERUSALEM LTD.
发明人:
申请号:
IL14337901
公开号:
IL143379A
申请日:
2001.05.24
申请国别(地区):
IL
年份:
2013
代理人:
摘要:
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.