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人工合成的反义寡核苷酸抑制相关基因的转录和翻译，对许多涉及基因表达的疾病有一定的治疗价值。对反义寡核苷酸的药动学和药效学、不良反应已有较多临床前研究，其对肿瘤、病毒感染等的临床试验的初步结果，都显示了它的应用前景。
Synthetic antisense oligonucleotides inhibit the transcription and translation of related genes and have therapeutic value in many diseases involving gene expression. Antisense oligonucleotide pharmacokinetics and pharmacodynamics, adverse reactions have more preclinical studies, its preliminary results of clinical trials of tumors, viral infections, have shown its application prospects.