Exploring molecular genetics and gene editing approaches of peripheral neuropathies: A future treatment approach

IF 0.9 Q4 GENETICS & HEREDITY
Fatima Muzamil , Muhammad Waseem Sajjad
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引用次数: 0

Abstract

Peripheral neuropathies are conditions in which the nerves of the peripheral nervous system (PNS) become disrupted. A tingling sensation and apathy are frequent symptoms. These symptoms could be unpleasant and are usually followed by fatigue. The hallmarks of acquired peripheral neuropathy (APN) include metabolic issues, inflammatory reactions, and impairments in axonal communication. Presently, it is known that errors in genetic material that encode proteins with a wide variety of activities result in different forms of Charcot-Marie-Tooth diseases (CMT) and associated hereditary peripheral neuropathies. Among these are cytoskeletal proteins, adhesion molecules, gap-junction proteins, transcription factors, receptors for neurotropic factors, structural proteins related to forming compact myelin, proteins involved in signalling pathways, and proteins involved in concealing the cell division process. The prospects of developing effective medications for neuropathic pain depend on the potential to comprehend pain mechanisms. Molecular methods for treating neuropathic pain include RNAi and gene-based therapies. New methods of gene therapy for the management of Inherited peripheral neuropathic conditions have been made possible by the latest innovations in genetic modification technologies. Driven by both inherited and acquired causes, the increasing frequency of peripheral neuropathies calls for creative and exact molecular therapies. This study is particularly relevant as direct manipulation of neuropathy-related mutations made possible by modern gene editing technologies like CRISPR-Cas9 and prime editing now helps Development of next-generation treatments depends on an awareness of these techniques.
探索周围神经病变的分子遗传学和基因编辑方法:未来的治疗方法
周围神经病变是指周围神经系统(PNS)的神经受到破坏。刺痛感和冷漠是常见的症状。这些症状可能令人不愉快,并且通常伴随着疲劳。获得性周围神经病变(APN)的特征包括代谢问题、炎症反应和轴突通讯障碍。目前,已知编码多种活性蛋白的遗传物质中的错误会导致不同形式的沙科-玛丽- tooth病(CMT)和相关的遗传性周围神经病变。其中包括细胞骨架蛋白、粘附分子、间隙连接蛋白、转录因子、神经营养因子受体、与形成致密髓磷脂相关的结构蛋白、参与信号通路的蛋白质和参与隐藏细胞分裂过程的蛋白质。开发神经性疼痛有效药物的前景取决于理解疼痛机制的潜力。治疗神经性疼痛的分子方法包括RNAi和基于基因的治疗。遗传修饰技术的最新创新使遗传周围神经病变的基因治疗管理的新方法成为可能。由于遗传和获得性原因的驱动,周围神经病变的频率越来越高,需要创造性和精确的分子治疗。这项研究尤其重要,因为现代基因编辑技术(如CRISPR-Cas9和启动编辑)使直接操纵神经病变相关突变成为可能,这有助于下一代治疗方法的开发,这取决于对这些技术的认识。
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来源期刊
Gene Reports
Gene Reports Biochemistry, Genetics and Molecular Biology-Genetics
CiteScore
3.30
自引率
7.70%
发文量
246
审稿时长
49 days
期刊介绍: Gene Reports publishes papers that focus on the regulation, expression, function and evolution of genes in all biological contexts, including all prokaryotic and eukaryotic organisms, as well as viruses. Gene Reports strives to be a very diverse journal and topics in all fields will be considered for publication. Although not limited to the following, some general topics include: DNA Organization, Replication & Evolution -Focus on genomic DNA (chromosomal organization, comparative genomics, DNA replication, DNA repair, mobile DNA, mitochondrial DNA, chloroplast DNA). Expression & Function - Focus on functional RNAs (microRNAs, tRNAs, rRNAs, mRNA splicing, alternative polyadenylation) Regulation - Focus on processes that mediate gene-read out (epigenetics, chromatin, histone code, transcription, translation, protein degradation). Cell Signaling - Focus on mechanisms that control information flow into the nucleus to control gene expression (kinase and phosphatase pathways controlled by extra-cellular ligands, Wnt, Notch, TGFbeta/BMPs, FGFs, IGFs etc.) Profiling of gene expression and genetic variation - Focus on high throughput approaches (e.g., DeepSeq, ChIP-Seq, Affymetrix microarrays, proteomics) that define gene regulatory circuitry, molecular pathways and protein/protein networks. Genetics - Focus on development in model organisms (e.g., mouse, frog, fruit fly, worm), human genetic variation, population genetics, as well as agricultural and veterinary genetics. Molecular Pathology & Regenerative Medicine - Focus on the deregulation of molecular processes in human diseases and mechanisms supporting regeneration of tissues through pluripotent or multipotent stem cells.
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