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Genome Editing: A Practical Guide to Research and Clinical Applications [Mīkstie vāki]

(Associate Professor of Cardiovascular Medicine and Genetics, Perelman School of Medicine, University of Pennsylvania, USA)
  • Formāts: Paperback / softback, 230 pages, height x width: 235x191 mm, weight: 410 g, 75 illustrations (25 in full color); Illustrations, unspecified
  • Izdošanas datums: 10-Mar-2021
  • Izdevniecība: Academic Press Inc
  • ISBN-10: 0128234849
  • ISBN-13: 9780128234846
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  • Cena: 167,86 €
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Genome Editing: A Practical Guide to Research and Clinical ApplicationsPalielināt
  • Formāts: Paperback / softback, 230 pages, height x width: 235x191 mm, weight: 410 g, 75 illustrations (25 in full color); Illustrations, unspecified
  • Izdošanas datums: 10-Mar-2021
  • Izdevniecība: Academic Press Inc
  • ISBN-10: 0128234849
  • ISBN-13: 9780128234846
Citas grāmatas par šo tēmu:
Permanent link: https://www.kriso.lv/db/9780128234846.html
Keywords:

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.

  • Help readers familiarise with the variety of genome-editing approaches that are being applied in cardiovascular research and medicine, i.e., both research applications and clinical applications
  • Understand the use of genome editing through worked examples (based on real-life experiments) in which CRISPR-Cas9 is employed, online tools to design CRISPR-Cas9 reagents, methods to interpret data from genome-editing experiments, the downsides of genome-editing technology - both the scientific and ethical pitfalls to avoid
  • Written in an easy-to-follow manner, guiding readers from the design of the project to its completion
  • Includes unpublished and new methods
Preface xi
1 A brief history and primer on genome editing
1(20)
Homologous recombination
1(2)
Zinc-finger nucleases (ZFNs)
3(5)
Transcription activator-like effector nucleases (TALENs)
8(4)
CRISPR-Cas9 and RNA-guided endonucleases
12(4)
References
16(5)
2 Choosing a genome editing strategy and target site
21(20)
Introduction
21(1)
Gene knockout with single-site targeting
21(6)
Gene knockout with double-site targeting
27(4)
Gene knockout via sequence insertion, and the problem of long noncoding RNAs
31(3)
Inserting or correcting mutations
34(2)
Inserting a gene or other DNA sequence
36(2)
References
38(3)
3 Choosing a nuclease, guide RNA, and repair template
41(20)
Streptococcus pyogenes CRISPR-Cas9
41(3)
Choosing Streptococcus pyogenes Cas9 guide RNAs for non-homologous end joining
44(5)
Choosing guide RNAs and synthetic DNA repair templates for homology-directed repair
49(3)
Streptococcus pyogenes Cas9 with altered protospacer-adjacent motif preferences
52(1)
Alternative Cas9 proteins
53(2)
Cas12 proteins and other potential editors
55(3)
Afternote
58(1)
References
58(3)
4 Assessing the outcomes of genome editing
61(20)
Methods to assess genome editing of bulk cells
61(6)
Methods to assess genome editing of clonal cells
67(6)
Mosaicism
73(4)
Assessing for homology-directed repair and ruling out hemizygosity
77(3)
References
80(1)
5 Assessing for off-target mutagenesis
81(20)
The potential hazards of off-target mutagenesis
81(1)
Defining the extent of off-target mutagenesis
82(4)
Discovery phase
86(5)
Validation phase
91(1)
Strategies to reduce off-target mutagenesis
92(4)
Practical example
96(2)
References
98(3)
6 Base editing
101(22)
The BE(x) cytosine base editors
101(5)
Other cytosine base editors
106(2)
Adenine base editors
108(1)
Mitochondrial base editors
109(1)
Off-target effects of base editors
110(3)
Base editing for gene knockout
113(3)
Base editing for inserting or correcting mutations
116(3)
References
119(4)
7 Alternative types of editing
123(22)
Epigenome editing
123(3)
Practical example of epigenome editing
126(3)
Genome-wide CRISPR screens
129(2)
RNA targeting and editing
131(4)
Practical example of RNA targeting
135(1)
Prime editing
136(3)
Practical example of prime editing
139(2)
References
141(4)
8 Genome editing for cellular disease modeling
145(24)
Human pluripotent stem cells and the advantages of genome editing
145(5)
Study design considerations: example #1
150(6)
Study design considerations: example #2
156(4)
Study design considerations: example #3
160(6)
Summary
166(1)
References
166(3)
9 Genome editing for functional experiments and screens
169(24)
Rapid generation of knockoutand knock-in mouse models: The study of KLF14
169(7)
Combining a variety of editing approaches: The study of two lipid-associated loci
176(6)
Multiplex genome editing: Generating an allelic series of cell lines to annotate patient variants of uncertain significance
182(4)
Genome-wide CRISPR screens: Improving hepatocyte differentiation and identifying genetic modifiers of doxorubicin-induced cardiotoxicity
186(4)
References
190(3)
10 Therapeutic genome editing
193(20)
Ex vivo therapeutic genome editing
193(1)
In vivo therapeutic genome editing
194(9)
Practical example
203(4)
Ethical considerations
207(2)
References
209(4)
Index 213
Kiran Musunuru, MD, PhD, MPH, ML received his medical degree from Weill Cornell Medical College, his PhD from The Rockefeller University, and his Master of Public Health from Johns Hopkins Bloomberg School of Public Health. He trained in Internal Medicine at Brigham and Womens Hospital and Cardiovascular Medicine at Johns Hopkins Hospital, followed by postdoctoral work at Massachusetts General Hospital and the Broad Institute of MIT and Harvard. Dr Musunurus research focuses on the genetics of cardiovascular and metabolic diseases and seeks to identify naturally occurring genetic variants that predispose to or protect against disease and can be used to develop therapies to protect the entire population. His expertise includes the use of human pluripotent stem cells as a platform for disease modelling and the use of genome-editing tools such as CRISPR-Cas9 for research and therapeutic applications. In 2016, he received the Presidential Early Career Award for Scientists and Engineers from U.S. President Obama, as well as the American Heart Associations Award of Meritorious Achievement. He became Editor-in-Chief of Circulation: Genomic and Precision Medicine (an American Heart Association journal) in 2018.