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Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
专利权人:
BIOMARIN TECHNOLOGIES B.V.
发明人:
DE VISSER PETER CHRISTIAN,MULDERS SUSAN ALLEGONDA MARIA
申请号:
NZ70056113
公开号:
NZ700561A
申请日:
2013.04.23
申请国别(地区):
NZ
年份:
2016
代理人:
摘要:
The current invention provides an improved oligonucleotide consisting of a 2&rsquo-O-methyl RNA nucleotide residue, having a backbone wherein all phosphate moieties are replaced by phosphorothioate moieties, having a base sequence consisting of (XYG)7 wherein each X is 5-methylcytosine and each Y is Uracil and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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