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RNA interference (RNAi) is a widely used technology for gene silencing and has become a key tool in a myriad of research and lead discoveries. In recent years, the mechanism of RNAi agents has been well investigated, and the technique has been optimized for better effectiveness and safety. On the other hand, the clustered regularly interspaced short palindromic repeats (CRISPR)-associated Cas9/gRNA system is a recent, novel, targeted genome-editing technique derived from the bacterial immune system. Recent advances in gene-editing research and technologies have enabled the CRISPR Cas9 system to become a popular tool for sequence-specific gene editing to correct and modify eukaryotic systems. In this book, we will focus on the mechanisms, applications, regulations (their pros and cons), and various ways in which RNAi-based methods and CRIPSR-Cas9 technology have stimulated the modulation of gene expression, thereby making them a promising therapeutic tool to treat and prevent complex diseases and disorders.

CRISPR-associated protein 9. --- Cas9 (CRISPR-associated protein 9) --- CRISPR/Cas9 --- Endonucleases --- Gene editing --- Life Sciences --- Molecular Genetics --- Genetics and Molecular Biology --- Genomics --- Biochemistry

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Would you change your genes if you could? As we confront the 'industrial revolution of the genome', the recent discoveries of Crispr-Cas9 technologies are offering, for the first time, cheap and effective methods for editing the human genome. This opens up startling new opportunities as well as significant ethical uncertainty. Tracing events across a fifty-year period, from the first gene splicing techniques to the present day, this is the story of gene editing - the science, the impact and the potential. Kozubek weaves together the fascinating stories of many of the scientists involved in the development of gene editing technology. Along the way, he demystifies how the technology really works and provides vivid and thought-provoking reflections on the continuing ethical debate. Ultimately, Kozubek places the debate in its historical and scientific context to consider both what drives scientific discovery and the implications of the 'commodification' of life.

Gene targeting. --- Genomes. --- CRISPR-associated protein 9. --- Cas9 (CRISPR-associated protein 9) --- CRISPR/Cas9 --- Endonucleases --- Gene editing --- Genetics --- Genomics --- Haploidy --- Targeting, Gene --- Genetic engineering

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Genome Editing: A Practical Guide to Research and Clinical Applications is geared towards investigators interested in learning how to use CRISPR-Cas9-based technologies, with a focus on cardiovascular research and clinical applications. Covering a range of topics from the basics of genome editing to design considerations, to assessments and applications, this reference allows readers to get started and establish a full workflow from the beginning of the project to its full completion. With worked examples drawn from real-life experiments, as well as troubleshooting and pitfalls to avoid, the book serves as an essential reference for researchers and investigators in both cardiovascular and biomedical research.

Gene editing. --- CRISPR-associated protein 9. --- CRISPR (Genetics). --- Cardiovascular system --- Diseases --- Genetic aspects. --- Circulatory system --- Vascular system --- Blood --- Clustered Regularly Interspaced Short Palindromic Repeats (Genetics) --- Immunogenetics --- Nucleotide sequence --- Cas9 (CRISPR-associated protein 9) --- CRISPR/Cas9 --- Endonucleases --- Gene editing --- Editing, Gene --- Editing, Genome --- Genome editing --- Genetic engineering --- Circulation --- Methodology.