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Gene Therapy for Advanced Melanoma: Selective Targeting and Therapeutic Nucleic Acids

机译:晚期黑素瘤的基因治疗:选择性靶向和治疗性核酸

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摘要

Despite recent advances, the treatment of malignant melanoma still results in the relapse of the disease, and second line treatment mostly fails due to the occurrence of resistance. A wide range of mutations are known to prevent effective treatment with chemotherapeutic drugs. Hence, approaches with biopharmaceuticals including proteins, like antibodies or cytokines, are applied. As an alternative, regimens with therapeutically active nucleic acids offer the possibility for highly selective cancer treatment whilst avoiding unwanted and toxic side effects. This paper gives a brief introduction into the mechanism of this devastating disease, discusses the shortcoming of current therapy approaches, and pinpoints anchor points which could be harnessed for therapeutic intervention with nucleic acids. We bring the delivery of nucleic acid nanopharmaceutics into perspective as a novel antimelanoma therapeutic approach and discuss the possibilities for melanoma specific targeting. The latest reports on preclinical and already clinical application of nucleic acids in melanoma are discussed.
机译:尽管有最近的进展,但是恶性黑色素瘤的治疗仍然导致疾病的复发,并且二线治疗主要是由于耐药性的出现而失败。已知多种突变会阻止化学疗法药物的有效治疗。因此,应用了包括蛋白质的生物药物的方法,例如抗体或细胞因子。作为替代方案,具有治疗活性核酸的方案为高度选择性的癌症治疗提供了可能性,同时避免了不良和毒性的副作用。本文简要介绍了这种毁灭性疾病的机理,讨论了当前治疗方法的不足,并指出了可用于核酸治疗干预的定位点。我们将核酸纳米药物的交付作为一种新的抗色素沉着瘤的治疗方法进行了展望,并讨论了针对黑色素瘤的特异性靶向方法的可能性。讨论了有关核酸在黑色素瘤中的临床前和临床应用的最新报道。

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