دورية أكاديمية
Huntingtin and Its Role in Mechanisms of RNA-Mediated Toxicity
| العنوان: | Huntingtin and Its Role in Mechanisms of RNA-Mediated Toxicity |
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| المؤلفون: | Annika Heinz, Deepti Kailash Nabariya, Sybille Krauss |
| المصدر: | Toxins, Vol 13, Iss 7, p 487 (2021) |
| بيانات النشر: | MDPI AG, 2021. |
| سنة النشر: | 2021 |
| المجموعة: | LCC:Medicine |
| مصطلحات موضوعية: | huntingtin, RNA toxicity, CAG repeat, neurodegeneration, RNA hairpin, RNA binding protein (RBP), Medicine |
| الوصف: | Huntington’s disease (HD) is caused by a CAG-repeat expansion mutation in the Huntingtin (HTT) gene. It is characterized by progressive psychiatric and neurological symptoms in combination with a progressive movement disorder. Despite the ubiquitous expression of HTT, pathological changes occur quite selectively in the central nervous system. Since the discovery of HD more than 150 years ago, a lot of research on molecular mechanisms contributing to neurotoxicity has remained the focal point. While traditionally, the protein encoded by the HTT gene remained the cynosure for researchers and was extensively reviewed elsewhere, several studies in the last few years clearly indicated the contribution of the mutant RNA transcript to cellular dysfunction as well. In this review, we outline recent studies on RNA-mediated molecular mechanisms that are linked to cellular dysfunction in HD models. These mechanisms include mis-splicing, aberrant translation, deregulation of the miRNA machinery, deregulated RNA transport and abnormal regulation of mitochondrial RNA. Furthermore, we summarize recent therapeutical approaches targeting the mutant HTT transcript. While currently available treatments are of a palliative nature only and do not halt the disease progression, recent clinical studies provide hope that these novel RNA-targeting strategies will lead to better therapeutic approaches. |
| نوع الوثيقة: | article |
| وصف الملف: | electronic resource |
| اللغة: | English |
| تدمد: | 13070487 2072-6651 |
| Relation: | https://www.mdpi.com/2072-6651/13/7/487; https://doaj.org/toc/2072-6651 |
| DOI: | 10.3390/toxins13070487 |
| URL الوصول: | https://doaj.org/article/d0f0df984bed4329905a3b8cc79e76ab |
| رقم الأكسشن: | edsdoj.0f0df984bed4329905a3b8cc79e76ab |
| قاعدة البيانات: | Directory of Open Access Journals |
Full Text Finder | تدمد: | 13070487 20726651 |
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| DOI: | 10.3390/toxins13070487 |