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Gene Silencing and Editing Strategies for Neurodegenerative Diseases

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Book Series: Frontiers Research Topics ISSN: 16648714 ISBN: 9782889455515 Year: Pages: 115 DOI: 10.3389/978-2-88945-551-5 Language: English
Publisher: Frontiers Media SA
Subject: Science (General) --- Neurology
Added to DOAB on : 2019-01-23 14:53:42
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Abstract

Neurodegenerative diseases (NDs) are a heterogeneous group of disorders affecting the central nervous system. Despite significant differences in their causes, neuropathological abnormalities, and clinical outcomes, some similarities can be found among them, as for example: 1) frequent aggregation and deposition of misfolded proteins, 2) common molecular mechanisms leading to neurodegeneration, and 3) certain overlap in symptoms and clinical features. To date, there is no cure that could stop or delay the progression of these diseases. The advent of advanced gene therapy techniques such as gene silencing and gene editing opened a new avenue for the development of therapeutic strategies for NDs.The discovery of the RNA interference (RNAi) mechanism, in 1998, by Andrew Fire and Craig Mello allowed an important boost to the gene therapy field, providing a potential therapeutic strategy to treat inherited dominant genetic disorders. The use of small RNA sequences to control the expression of disease-causing genes rapidly implemented in the preclinical studies for different diseases. In the field of NDs, several successful studies using this technology proved its potential as a therapeutic option. However, issues like the type of delivery system (non-viral versus viral) or the potential toxicity of the small RNA molecules, made the translation of gene silencing therapeutics to human application very slow and difficult.Recently, a new hope in the gene therapy field emerged with the development of gene editing techniques like TALENs or CRISPR/Cas9 systems. The opportunity of editing or deleting gene sequences drove the scientific community euphoric, with an enormous increase in the number of published studies using this type of techniques. Recently, the first clinical trial using one of these systems was approved in China. For NDs, gene-editing technology also represents an important therapeutic option, and the first preclinical studies are now being published, showing the potential accomplishment for this technology.

Regulatory microRNA

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ISBN: 9783038977681 9783038977698 Year: Pages: 348 DOI: 10.3390/books978-3-03897-769-8 Language: eng
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Science (General) --- Biology --- Genetics
Added to DOAB on : 2019-04-25 16:37:17
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Abstract

This book includes updated information about microRNA regulation, for example, in the fields of circular RNAs, multiomics analysis, biomarkers and oncogenes. The variety of topics included in this book reaffirms the extent to which microRNA regulation affects biological processes. Although microRNAs are not translated to proteins, their importance for biological processes is not less than proteins. An understanding of their roles in various biological processes is critical to understanding gene function in these biological processes. Although non-coding RNAs other than microRNAs have recently come under investigation, microRNA still remains the front runner as the subject of genetic and biological studies. In reading the collection of papers, readers can grasp the most updated information regarding microRNA regulation, which will continue to be an important topic in genetics and biology.

Keywords

tensor decomposition --- miRNA transfection --- sequence-nonspecific off-target regulation --- extracellular vesicles --- cancer --- therapeutics --- miRNA --- virus --- host --- Cross-Kingdom --- target prediction --- microRNAs --- autophagy --- mitophagy --- cardiac diseases --- biomarker --- calf --- Ileum --- miRNA-mRNA integration --- miRNA sequencing --- growth --- development --- microRNA --- myelodysplastic syndromes --- acute myeloid leukemia --- azacitidine --- 14q32 --- MEG3 --- autophagy regulator --- transcriptional factor --- non-coding RNA --- regulatory network --- RWR algorithm --- circular RNA --- circFGFR2 --- FGFR2 --- miR-133a-5p --- miR-29b-1-5p --- skeletal muscle --- proliferation --- differentiation --- breast cancer --- CAFs --- estrogens --- GPER --- miR-338-3p --- c-Fos --- Cyclin D1 --- amyotrophic lateral sclerosis (ALS) --- biomarker --- microRNA --- cerebrospinal fluid (CSF) --- muscle biopsy --- circulating miRNAs --- RNA interference --- small interfering RNA --- microRNA --- oncolytic virotherapy --- conditionally replicating adenovirus (CRAd) --- biomarker --- gene --- microRNA --- parkinson’s disease --- miRNA --- bioinformatic analysis --- ischemic stroke --- miRNA-gene target interaction --- network --- biomarker --- diagnosis --- prognosis --- microRNAs --- epigenetic biomarker --- sepsis --- inflammation --- Teleostei --- embryogenesis --- tissue-enriched miRNAs --- post-transcriptional gene regulation --- miRNA expression and regulation --- passenger miRNA --- biomarker --- vascular injury --- smooth muscle cells --- porcine vein graft and stent models --- bone angiogenesis --- osteogenesis --- angiogenic-osteogenic coupling --- microRNAs --- bone regeneration --- bone formation --- bone tissue-engineering --- angiomiRs --- osteomiRs --- hypoxamiRs --- circular RNA --- circHIPK3 --- microRNA --- miR-30a-3p --- skeletal muscle --- proliferation --- differentiation

Transcriptional Regulation: Molecules, Involved Mechanisms and Misregulation

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ISBN: 9783039212651 / 9783039212668 Year: Pages: 356 DOI: 10.3390/books978-3-03921-266-8 Language: eng
Publisher: MDPI - Multidisciplinary Digital Publishing Institute
Subject: Science (General) --- Biology
Added to DOAB on : 2019-08-28 11:21:27
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Abstract

Transcriptional regulation is a critical biological process involved in the response of a cell, a tissue or an organism to a variety of intra- and extra-cellular signals. Besides, it controls the establishment and maintenance of cell identity throughout developmental and differentiation programs. This highly complex and dynamic process is orchestrated by a huge number of molecules and protein networks and occurs through multiple temporal and functional steps. Of note, many human disorders are characterized by misregulation of global transcription since most of the signaling pathways ultimately target components of transcription machinery. This book includes a selection of papers that illustrate recent advances in our understanding of transcriptional regulation and focuses on many important topics, from cis-regulatory elements to transcription factors, chromatin regulators and non-coding RNAs, other than several transcriptome studies and computational analyses.

Keywords

major depressive disorder --- glioblastoma --- differentially expressed genes --- transcriptomics --- common pathway --- mouse --- miR-25-3p --- Akt1 --- AP-2? --- promoter --- cell metabolism --- p57Kip2 --- CDKN1C --- epigenetics --- disease --- cell differentiation --- placenta --- long non-coding RNA (lncRNA) --- human --- pregnancy --- high-throughput RNA sequencing (RNA-Seq) --- transcriptome --- Rsh regulon --- Novosphingobium pentaromativorans US6-1 --- sphingomonads --- RNA-seq --- N-acyl-l-homoserine lactone --- ppGpp --- selenium --- selenocysteine --- selenoproteins --- selenocysteine insertion sequence --- nonsense-mediated decay --- G-quadruplex --- transcriptional regulation --- promoter --- CRISPR/Cas9 --- PRDM gene family --- TCGA data analysis --- somatic mutations --- transcriptome profiling --- human malignancies --- tristetraprolin (TTP) --- tumorigenesis --- posttranscriptional regulation --- adenosine and uridine-rich elements (AREs) --- circRNA-disease associations --- pathway --- heterogeneous network --- Patau Syndrome --- cytogenetics --- FOXO1 --- transcription factor --- molecular pathways --- bioinformatics --- molecular docking --- and drug design --- transcription regulation --- gene expression --- causal inference --- enhancer activity --- insect --- transcription factors --- structures and functions --- research methods --- progress and prospects --- Pax3 --- Pteria penguin (Röding, 1798) --- tyrosinase --- melanin --- RNA interference --- liquid chromatograph-tandem mass spectrometer (LC-MS/MS) --- epigenetics --- gene expression --- nutrition --- transcription --- disorders --- mechanisms --- Crassostrea gigas --- Pacific oyster --- pediveliger larvae --- bioadhesive --- transcriptome --- gene expression --- interactome --- microscopy --- fertilization --- self-incompatibility --- transcriptome --- tea --- long non-coding RNAs --- cancer --- acute leukemia --- therapeutic targets --- Adiponectin --- cancer --- Adiponectin receptors --- obesity --- inflammatory response --- inflammation --- nutritional status --- n/a

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