Getting miRNA Therapeutics into the Target Cells for Neurodegenerative Diseases: A Mini-Review
作者： Ming Ming Wen
关键词: Gene 、 Gene silencing 、 microRNA 、 Amyotrophic lateral sclerosis 、 Gene expression 、 Bioinformatics 、 Function (biology) 、 RNA interference 、 Gene knockdown 、 Biology
摘要: Abstract: MiRNAs play important roles in modulating gene expression varying cellular processes and disease pathogenesis, including neurodegenerative diseases. Several miRNAs are expressed the brain control development identified as biomarkers pathogenesis of motor- neuro-cognitive diseases such Alzheimer, Huntington's Parkinson's amyotrophic lateral sclerosis. These remarkable could be used diagnostic markers therapeutic targeting potential for many stressful untreatable progressive To modulate these miRNA activities, there currently two strategies involved; first one is to therapeutically restore suppressed level by mimics (agonist), other inhibit function using antimiR (antagonist) repress overactive function. However, RNAi-based therapeutics often faces vivo instability because naked nucleic acids subject enzyme degradation before reaching target sites. Therefore, an effective, safe stable bio-responsive delivery system necessary protect from serum assist their entrance cells. Since neuronal cells non-regenerating, design engineered delivered CNS long term knockdown representing enormous challenge scientists. This article provides insight summary on some innovative employed deliver into viral non-viral carrier systems hold promise RNA therapy
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