Modulation of gene expression by antisense and antigene oligodeoxynucleotides and small interfering RNA.
Modulation of gene expression by antisense and antigene oligodeoxynucleotides and small interfering RNA.
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DOI：
10.1517/17425247.2.1.3
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发表时间：
2005-01-01
影响因子：
6.6
通讯作者：
Guntaka, Ramareddy V
中科院分区：
文献类型：
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作者：
Mahato, Ram I;Cheng, Kun;Guntaka, Ramareddy V
反义寡核苷酸、三链形成寡核苷酸和双链小干扰RNA在治疗许多严重和衰弱的疾病方面具有巨大的潜力。产业界和学术界的共同努力，在将这些核酸药物转化为治疗药物方面取得了重大进展，临床上已经有一种FDA批准的反义药物。尽管一种产品和其他几种正在进行的临床试验取得了成功，但在它们的稳定性、细胞摄取、处置、特定部位给药和治疗效果方面仍然存在挑战。综述了这些核酸的原理、策略和传递考虑。此外，还讨论了克服生物障碍的方法，以便在目标部位的治疗浓度可以维持一段所需的时间。
Antisense oligodeoxynucleotides, triplex-forming oligodeoxynucleotides and double-stranded small interfering RNAs have great potential for the treatment of many severe and debilitating diseases. Concerted efforts from both industry and academia have made significant progress in turning these nucleic acid drugs into therapeutics, and there is already one FDA-approved antisense drug in the clinic. Despite the success of one product and several other ongoing clinical trials, challenges still exist in their stability, cellular uptake, disposition, site-specific delivery and therapeutic efficacy. The principles, strategies and delivery consideration of these nucleic acids are reviewed. Furthermore, the ways to overcome the biological barriers are also discussed so that therapeutic concentrations at their target sites can be maintained for a desired period.