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RNA干扰与细胞外囊泡联合治疗脊髓小脑共济失调

Combinational treatments of RNA interference and extracellular vesicles in the spinocerebellar ataxia.

作者信息

Ding Yingying, Zhang Yong, Liu Xuehong

机构信息

Department of Histology and Embryology, Medical College, Shaoxing University, Shaoxing, Zhejiang, China.

Department of Clinical Medicine, Affiliated Hospital of Hangzhou Normal University, Hangzhou, China.

出版信息

Front Mol Neurosci. 2022 Oct 13;15:1043947. doi: 10.3389/fnmol.2022.1043947. eCollection 2022.

Abstract

Spinocerebellar ataxia (SCA) is an autosomal dominant neurodegenerative disease (ND) with a high mortality rate. Symptomatic treatment is the only clinically adopted treatment. However, it has poor effect and serious complications. Traditional diagnostic methods [such as magnetic resonance imaging (MRI)] have drawbacks. Presently, the superiority of RNA interference (RNAi) and extracellular vesicles (EVs) in improving SCA has attracted extensive attention. Both can serve as the potential biomarkers for the diagnosing and monitoring disease progression. Herein, we analyzed the basis and prospect of therapies for SCA. Meanwhile, we elaborated the development and application of miRNAs, siRNAs, shRNAs, and EVs in the diagnosis and treatment of SCA. We propose the combination of RNAi and EVs to avoid the adverse factors of their respective treatment and maximize the benefits of treatment through the technology of EVs loaded with RNA. Obviously, the combinational therapy of RNAi and EVs may more accurately diagnose and cure SCA.

摘要

脊髓小脑共济失调(SCA)是一种常染色体显性神经退行性疾病(ND),死亡率很高。对症治疗是目前临床上唯一采用的治疗方法。然而,其疗效不佳且并发症严重。传统的诊断方法[如磁共振成像(MRI)]存在缺陷。目前,RNA干扰(RNAi)和细胞外囊泡(EVs)在改善SCA方面的优势已引起广泛关注。两者都可作为诊断和监测疾病进展的潜在生物标志物。在此,我们分析了SCA治疗的基础和前景。同时,阐述了微小RNA(miRNAs)、小干扰RNA(siRNAs)、短发夹RNA(shRNAs)和细胞外囊泡(EVs)在SCA诊断和治疗中的发展及应用。我们提出将RNAi和EVs联合使用,以避免各自治疗的不利因素,并通过装载RNA的EVs技术使治疗效益最大化。显然,RNAi与EVs的联合治疗可能会更准确地诊断和治愈SCA。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/42c6/9606576/941e9d3df70e/fnmol-15-1043947-g001.jpg

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