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Biomedical technology. --- Gene silencing. --- Rna interference. --- Small interfering rna.

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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.

Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells --- Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells

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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.

Gene silencing --- Long non-coding RNAs --- RNA interference --- Neurodegenerative diseases --- CRISPR/Cas9 --- Neurodegeneration --- Gene editing --- Antisense oligonucleotides --- Neuroinflammation --- iPS cells

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The study of RNA interference, editing, and modification has led to major shifts in our understanding of how genes are expressed and regulated. In RNA Interference, Editing, and Modification: Methods and Protocols, hands-on experimentalists describe in detail the protocols and assays they have developed to study these processes in most of the major biological systems in which they are known to occur-a wide range of organisms that includes worms, flies, trypanosomes, mammals, and plants. A historical overview of each subject is provided to put related chapters into a larger context. RNA interference (RNAi) has also emerged in recent years as an important and promising tool for gene discovery and reverse genetic engineering. Application of this cutting-edge technique is described for a range of experimental systems. Topics of interest include stable and transient RNA interference, RNA editing, gene silencing, bioinformatics, small noncoding RNAs, and RNomics. Special attention is given to methods for the identification and characterization of small RNAs, many of which are involved in RNA interference or modification. In addition, techniques used to study RNA editing mechanisms are elaborated for both systems that involve the conversion of one base to another and insertion-deletion editing. To promote the adaptation of their assays and approaches for use in other biological systems, the authors have included a range of methods representative of the major experimental systems in a given field. All protocols presented follow the successful Methods in Molecular Biology™ series format, each one offering step-by-step laboratory instructions, an introduction outlining the principle behind the technique, lists of equipment and reagents, and tips on troubleshooting and avoiding known pitfalls. State-of-the-art and highly practical, RNA Interference, Editing, and Modification: Methods and Protocols offers not only geneticists, molecular biologists, and bioinformaticists a comprehensive collection of readily reproducible techniques for the elucidation of gene function and the alteration of gene expression, but also describes how best they may be productively used to address outstanding mechanistic questions and adapted to novel biological systems.

RNA Editing --- RNA Interference --- RNA editing --- ARN --- Laboratory manuals. --- Edition --- Manuels de laboratoire --- RNA editing -- Laboratory manuals. --- Laboratory Manuals --- Publication Formats --- RNA Processing, Post-Transcriptional --- Gene Silencing --- Epigenesis, Genetic --- Biochemical Processes --- Publication Characteristics --- Metabolism --- Gene Expression Regulation --- Genetic Processes --- Chemical Processes --- Metabolic Phenomena --- Biochemical Phenomena --- Genetic Phenomena --- Chemical Phenomena --- Phenomena and Processes --- Genetics --- Biology - General --- Biochemistry --- Biology --- Chemistry --- Health & Biological Sciences --- Physical Sciences & Mathematics --- Editing, RNA --- Messenger RNA editing --- mRNA editing --- Genetic regulation --- Biochemistry. --- Biochemistry, general. --- Biological chemistry --- Chemical composition of organisms --- Organisms --- Physiological chemistry --- Medical sciences --- Composition --- Genetic regulation. --- Genetic Phenomena. --- Rna editing --- Rna interference

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The critically acclaimed laboratory standard, Methods in Enzymology, is one of the most highly respected publications in the field of biochemistry. Since 1955, each volume has been eagerly awaited, frequently consulted, and praised by researchers and reviewers alike. The series contains much material still relevant today - truly an essential publication for researchers in all fields of life sciences.RNA Interference will cover RNAi in non-vertebrates (plants, C. elegans, drosophila, and S. pombe), and Mammalian systems (human and non-human cells). This volume discusses e

RNA Interference. --- Small interfering RNA. --- RNA Interference --- RNA, Small Interfering --- Gene Silencing --- Epigenesis, Genetic --- RNA, Antisense --- RNA, Small Untranslated --- RNA --- Antisense Elements (Genetics) --- RNA, Untranslated --- Gene Expression Regulation --- Nucleic Acids, Nucleotides, and Nucleosides --- Genetic Processes --- Nucleic Acids --- Genetic Phenomena --- Chemicals and Drugs --- Phenomena and Processes --- RNA editing --- Gene silencing --- Small interfering RNA --- piRNA (Piwi-interacting RNA) --- Piwi-interacting RNA --- Piwi protein-interacting RNA --- rasiRNA (Repeat-associated small interfering RNA) --- Repeat-associated siRNA --- Repeat-associated small interfering RNA --- Scan RNA --- scnRNA (Small scan RNA) --- Short hairpin RNA --- Short interfering RNA --- shRNA (Short hairpin RNA) --- siRNA (Small interfering RNA) --- Small hairpin RNA --- Small scan RNA --- tasiRNA (Trans-acting small interfering RNA) --- Trans-acting siRNA --- Trans-acting small interfering RNA --- Gene inactivation --- Inactivation, Gene --- Silencing, Gene --- Editing, RNA --- Messenger RNA editing --- mRNA editing --- Ribonucleic acid --- Ribose nucleic acid --- Antisense RNA --- Genetic regulation --- Nucleic acids --- Ribose