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Pharmaceutical composition comprising an antisense oligonucleotide and how to use it
Pharmaceutical composition comprising an antisense oligonucleotide and how to use it
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机译:包含反义寡核苷酸的药物组合物及其使用方法
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摘要
The invention provides compositions and associated methods for the antisense treatment of genetic disorders, infections and various other medical conditions. In particular, embodiments of the present invention are directed to pharmaceutical compositions comprising a combination of an antisense oligonucleotide compound conjugated with a positively charged polymer (ON-PCP) and a negatively charged polymer. Pharmaceutical compositions in accordance with the present invention have demonstrated improved antisense efficiency and reductions in cell toxicity compared to compositions that contain an oligonucleotide compound conjugated with a positively charged polymer.
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