Biodegradable polymers in gene-silencing technology
Biodegradable polymers in gene-silencing technology
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DOI：
10.1002/pat.4713
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发表时间：
2019-10-01
影响因子：
3.4
通讯作者：
Domb, Abraham J.
中科院分区：
文献类型：
--
作者：
Hosseinkhani, Hossein;Domb, Abraham J.
小干扰RNA（siRNA）技术已经显示出作为用于治疗癌症和其他疾病的一类新的治疗发明的巨大前景。siRNA已广泛用于阻断各种基因，目前正在评估其作为癌症和病毒性疾病的治疗剂。尽管序列特异性基因调控的这一显著生物学过程令人兴奋，但使用基于siRNA的治疗剂的主要限制是它们被血清核酸酶快速降解、细胞摄取差、全身递送后快速肾清除、脱靶效应和诱导免疫应答。许多研究人员试图克服这些限制，开发抗核酸酶的化学修饰的siRNA和各种合成和天然可生物降解的脂质和聚合物用于siRNA递送，以提高疗效和安全性。理想的基于siRNA的递送系统必须是临床上合适的、安全的和有效的。本文综述了近年来生物可降解聚合物在siRNA载体技术中的研究进展，小干扰RNA（siRNA）技术作为一种新型的治疗手段，在癌症和其他疾病的治疗中显示出巨大的应用前景。siRNA已广泛用于阻断各种基因，目前正在评估其作为癌症和病毒性疾病的治疗剂。尽管序列特异性基因调控的这一显著生物学过程令人兴奋，但使用基于siRNA的治疗剂的主要限制是它们被血清核酸酶快速降解、细胞摄取差、全身递送后快速肾清除、脱靶效应和诱导免疫应答。许多研究人员试图克服这些限制，开发抗核酸酶的化学修饰的siRNA和各种合成和天然可生物降解的脂质和聚合物用于siRNA递送，以提高疗效和安全性。理想的基于siRNA的递送系统必须是临床上合适的、安全的和有效的。本文综述了近年来生物可降解聚合物在siRNA载体技术中的研究进展。
Small interfering RNAs (siRNAs) technology has shown great promise as a new class of therapeutics invention for treatment of cancer and other diseases. siRNA has been used extensively in blocking various genes and is presently being evaluated as a therapeutic for cancer and viral disease. Despite the excitement about this remarkable biological process for sequence specific gene regulation, the major limitations against the use of siRNAs-based therapeutics are their rapid degradation by serum nuclease, poor cellular uptake, and rapid renal clearance following systemic delivery, off-target effects, and induction of immune responses. Many researchers have tried to overcome these limitations with developing nuclease-resistant chemically modified siRNAs and variety of synthetic and natural biodegradable lipids and polymers for siRNA delivery to enhance efficacy and safety profiles. An ideal siRNAs-based delivery system must be clinically suitable, safe, and effective. This review discuss the recent progress of biodegradable polymers in siRNA delivery technology.Small interfering RNAs (siRNAs) technology has shown great promise as a new class of therapeutics invention for treatment of cancer and other diseases. siRNA has been used extensively in blocking various genes and is presently being evaluated as a therapeutic for cancer and viral disease. Despite the excitement about this remarkable biological process for sequence specific gene regulation, the major limitations against the use of siRNAs-based therapeutics are their rapid degradation by serum nuclease, poor cellular uptake, and rapid renal clearance following systemic delivery, off-target effects, and induction of immune responses. Many researchers have tried to overcome these limitations with developing nuclease-resistant chemically modified siRNAs and variety of synthetic and natural biodegradable lipids and polymers for siRNA delivery to enhance efficacy and safety profiles. An ideal siRNAs-based delivery system must be clinically suitable, safe, and effective. This review discuss the recent progress of biodegradable polymers in siRNA delivery technology.