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首页> 外文期刊>Drug Target Insights >Recent Advancements in Targeted Delivery of Therapeutic Molecules in Neurodegenerative Disease - Spinocerebellar Ataxia - Opportunities and Challenges
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Recent Advancements in Targeted Delivery of Therapeutic Molecules in Neurodegenerative Disease - Spinocerebellar Ataxia - Opportunities and Challenges

机译：神经退行性疾病治疗性分子靶向递送的最新进展-脊髓小脑共济失调-机遇与挑战

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摘要

Drug discovery and its methodologies have been very effective in terms of treating cancers and immunological disorders but have not been able to stop genetic diseases as most of the drugs target at the protein level. They merely mitigate the symptoms of the disease. Spinocerebellar ataxia is a neurological genetic disorder that is caused by the formation of an abnormal protein. There have been several reports on ataxic drug development but actual clinical treatment is yet to be achieved. Oligonucleotide therapy called sequence specific siRNA mediated gene silencing has evolved with promising results. This approach emphasizes on suppressing the expression of the diseased gene at mRNA level. However, there is a limitation in delivery of siRNA to the target site. Several methods have been developed over the last decade to enhance the target specific delivery of DNA, siRNA, protein and small drug molecules for therapeutic purpose with less or no side effects. This review discusses the latest upcoming technologies in the field that focus on a number of nonviral nanocarriers for targeted delivery. In this review, we explore the promise and potential of novel therapeutics with interest on ataxia therapy.
机译：药物发现及其方法论在治疗癌症和免疫学疾病方面非常有效，但由于大多数药物都针对蛋白质水平，因此无法阻止遗传疾病。他们只是减轻疾病的症状。脊髓小脑共济失调是由异常蛋白质形成引起的一种神经遗传疾病。关于共济失调药物开发的报道已经很多，但实际的临床治疗尚待实现。称为序​​列特异性siRNA介导的基因沉默的寡核苷酸疗法已取得了可喜的成果。该方法强调在mRNA水平上抑制患病基因的表达。然而，在将siRNA递送至靶位点方面存在限制。在过去的十年中，已经开发了几种方法来增强DNA，siRNA，蛋白质和小分子药物的靶标特异性递送，从而达到治疗目的，而副作用很少或没有。这篇评论讨论了该领域中最新的技术，这些技术集中于多种非病毒纳米载体以进行靶向递送。在这篇综述中，我们探索了对共济失调疗法感兴趣的新型疗法的前景和潜力。

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