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El. knyga: CRISPR Genome Surgery in Stem Cells and Disease Tissues

Edited by (Institute for Genomic Medicine, Columbia University, New York, USA)
  • Formatas: PDF+DRM
  • Išleidimo metai: 20-Oct-2021
  • Leidėjas: Academic Press Inc
  • Kalba: eng
  • ISBN-13: 9780128178775
Kitos knygos pagal šią temą:
CRISPR Genome Surgery in Stem Cells and Disease TissuesDidesnis paveikslėlis
  • Formatas: PDF+DRM
  • Išleidimo metai: 20-Oct-2021
  • Leidėjas: Academic Press Inc
  • Kalba: eng
  • ISBN-13: 9780128178775
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CRISPR Genome Surgery in Stem Cells and Disease Tissues focuses uniquely on the clinical applications of CRISPR/Cas9 based technology. Topics include the latest advances in gene editing and its translational applications to various diseases, including retinal degenerative disease, recessively inherited diseases, and dominantly inherited diseases, to name a few. The book's target audience includes researchers, students, clinicians and the general public. This space that is not currently served by any existing resource, so this publication fills a gap in current literature.

  • Provides a thorough review of CRISPR-Cas9, from discovery to therapy
  • Covers the latest advances in gene editing and its translational applications to various diseases
  • Written by global leaders in the fields of gene editing and stem cell therapy
List of contributors
vii
Foreword ix
1 The history of CRISPR: from discovery to the present
Christine L. Xu
Stephen H. Tsang
The beginnings of CRISPR/Cas9
1(1)
Underlying mechanisms of CRISPR/Cas9
2(1)
Using CRISPR/Cas9 for mammalian gene editing
3(1)
Future directions
3(2)
Acknowledgments
5(1)
Author contributions
5(1)
Conflict of interest statement
5(1)
References
5(2)
2 CRISPR-Cas orthologs and variants
7(32)
Margaret E. Scollan
Introduction
7(1)
Cas9: a model of CRISPR-Cas systems
7(14)
CRISPR-Cas orthologs
21(7)
Conclusion
28(1)
References
28(11)
3 Genome engineering with CRISPR/Cas9, ZFNs, and TALENs
39(8)
Angela Y. Chang
Introduction
39(1)
Zinc-finger nucleases
40(1)
Transcription activator-like effector nucleases
41(1)
CRIPSR/Cas9
42(3)
Conclusion
45(1)
References
45(2)
4 Single-guide RNAs: rationale and design
47(10)
Angela Y. Chang
CRISPR/Cas9 background and the role of sgRNA
47(2)
Design of sgRNA
49(1)
Online tools to aid in sgRNA design
49(1)
Factors mediating sgRNA targeting specificity
49(1)
sgRNA design criteria to optimize specificity and efficiency while minimizing off-target activity
50(2)
sgRNA secondary structures impede formation of active Cas9---sgRNA complexes
52(1)
sgRNA and Cas9 concentration influences off-target activity
53(1)
Strategies to minimize sgRNA off-target activity
53(1)
Conclusion 54 References
54(3)
5 CRISPR-mediated dense mutagenesis: a tool for rational targeting of multiprotein complexes and the noncoding genome
57(8)
Falak Sher
Introduction
57(1)
Rational targeting of multiprotein complexes using CRISPR-mediated dense mutagenesis
58(1)
CRISPR-tiling screen workflow
59(1)
CRISPR-tiling library design, synthesis, and construction of lentivirus
59(2)
Lentiviral transduction and cell expansion
61(1)
Phenotype selection and next-generation sequencing
61(1)
Analysis of CRISPR-tiling screen
61(1)
Future directions and areas for improvement
62(1)
Conclusions
62(1)
References
62(3)
6 Targeting Alzheimer's disease and related dementias with CRISPR and human pluripotent stem cell technologies
65(16)
Andrew A. Sproul
Overview of AD and related dementias and how CRISPR technologies may help
65(2)
CRISPR for disease modeling genetic risk factors in hiPSCs
67(5)
CRISPR activation/inhibition/knock-out for modeling and genetic screens
72(2)
CRISPR for disease therapeutics
74(2)
Future directions
76(1)
References
77(4)
7 Gene editing for the cornea
81(20)
M. Andrew Nesbit
C.B. Tara Moore
The cornea
81(1)
Corneal dystrophies
81(2)
Gene therapy in the cornea
83(2)
CRISPR/Cas9 gene editing for corneal dystrophies
85(8)
Delivery of CRISPR/Cas9 gene editing components to the cornea
93(4)
Summary
97(1)
References
97(4)
8 Employing nonhomologous end joining and homology-directed repair for treatment of Leber congenital amaurosis and inherited retinal degeneration
101(10)
Dong Hyun Jo
Jin Hyoung Kim
Jeong Hun Kim
Leber congenital amaurosis and inherited retinal degeneration
101(1)
Genome surgery for treatment of human diseases
102(2)
Modes of correction with genome surgery
104(2)
Employing NHEJ and HDR for treatment of LCA and IRD
106(2)
Future direction and conclusions
108(1)
References
108(3)
9 The ethics of gene editing in human stem cells
111(12)
Alangoya Tezel
Overview of legal and political regulation
111(1)
Human embryonic stem cells
112(2)
Induced pluripotent stem cells
114(1)
Germline gene editing
115(3)
Looking ahead: the slippery slope
118(1)
References
119(4)
Index 123
Stephen H. Tsang is an acclaimed clinical geneticist in the care of individuals with retinal degenerations, and is known worldwide for his pivotal research in reprogramming the metabolome as a therapeutic avenue.

Dr. Tsang graduated from Johns Hopkins University, where he began his medical genetics training under the tutelage of Professor Victor A. McKusick. He received his M.D.-Ph.D. degrees from the NIH-National Institute of General Medical Sciences Medical Scientist Training Program (MSTP) at Columbia University. Dr. Tsang then completed his residency at Jules Stein Eye Institute/UCLA, followed by studies with Professors Alan C. Bird and Graham E. Holder on improving the care of individuals with macular degenerations.

Dr. Tsang is a recognized pioneer in genome surgery in stem cells. Most recently, he has been invited to lecture at the genome surgery workshop during the annual Association for Research in Vision & Ophthalmology (ARVO) 2015, 16 & 18 Annual Meetings; and as a Moderator for Gene Editing/Rewriting the Genome: Moving from Association to Biology and Therapeutics session during the 65th American Society of Human Genetics (ASHG) Annual Meeting, and a lecturer at 2015 & 2016 CRISPR Revolution conferences at Cold Spring Harbor.

In his New York State supported stem cell program (N09G-302), he is examining embryonic stem (ES) cells to model and replace diseased human retinal cells. His contributions were recognized by the 2005 Bernard Becker Association of University Professors in Ophthalmology's Research to Prevent Blindness Award. Dr. Tsang also participates in resident teaching and had been the Columbia ophthalmology basic science course director. He is a member of the American Society of Clinical Investigation.
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