首页> 外国专利> INHIBITING OR DOWNREGULATING GLYCOGEN SYNTHASE BY CREATING PREMATURE STOP CODONS USING ANTISENSE OLIGONUCLEOTIDES

INHIBITING OR DOWNREGULATING GLYCOGEN SYNTHASE BY CREATING PREMATURE STOP CODONS USING ANTISENSE OLIGONUCLEOTIDES

机译:使用反义寡核苷酸产生过早的止血密码子来抑制或下调糖原合成酶

摘要

The present disclosure relates to antisense oligonucleotides (AONs) for modulating the expression of glycogen synthase. AONs of the present disclosure may be useful in treating diseases associated with the modulation of the expression of the enzyme glycogen synthase, such as Pompe disease. Also provided by the present disclosure are compositions comprising AONs, as well as methods of down regulating mRNA coding for glycogen synthase, methods for reducing glycogen synthase in skeletal and cardiac muscle, and methods for treating Pompe disease.
机译:本公开涉及用于调节糖原合酶的表达的反义寡核苷酸(AORs)。本公开的AONS可用于治疗与调节酶糖原合酶的表达相关的疾病,例如POPPE疾病。本公开还提供了包含AORS的组合物,以及对糖原合酶编码的调节mRNA的方法,用于减少骨骼和心肌中的糖原合酶的方法,以及治疗Pompe疾病的方法。

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