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Crispr genome surgery in stem cells and disease tissues /
| Clasificación: | Libro Electrónico |
|---|---|
| Otros Autores: | |
| Formato: | Electrónico eBook |
| Idioma: | Inglés |
| Publicado: |
London :
Academic Press,
[2022]
|
| Temas: | |
| Acceso en línea: | Texto completo |
Tabla de Contenidos:
- Front Cover
- CRISPR Genome Surgery in Stem Cells and Disease Tissues
- Copyright Page
- Contents
- List of contributors
- Foreword
- 1 The history of CRISPR: from discovery to the present
- The beginnings of CRISPR/Cas9
- Underlying mechanisms of CRISPR/Cas9
- Using CRISPR/Cas9 for mammalian gene editing
- Future directions
- Acknowledgments
- Author contributions
- Conflict of interest statement
- References
- 2 CRISPR-Cas orthologs and variants
- Introduction
- Cas9: a model of CRISPR-Cas systems
- Nickase SpCas9
- High-fidelity and enhanced SpCas9
- SpCas9 variants with novel PAM requirements
- Deactivated-Cas9 characterization and application
- FokI-dCas9: a dCas9 application to improve specificity
- Base editing
- CRISPR-Cas orthologs
- CRISPR-Cas9 system orthologs
- Staphylococcus aureus Cas9
- Streptococcus thermophilus Cas9
- Neisseria meningitides Cas9
- Francisella novicida Cas9
- Campylobacter jejuni Cas9
- CRISPR-Cas12a (Cpf1) system
- CRISPR-Cas13a (C2c2) system
- Conclusion
- References
- 3 Genome engineering with CRISPR/Cas9, ZFNs, and TALENs
- Introduction
- Zinc-finger nucleases
- Transcription activator-like effector nucleases
- CRIPSR/Cas9
- Conclusion
- References
- 4 Single-guide RNAs: rationale and design
- CRISPR/Cas9 background and the role of sgRNA
- Design of sgRNA
- Online tools to aid in sgRNA design
- Factors mediating sgRNA targeting specificity
- sgRNA design criteria to optimize specificity and efficiency while minimizing off-target activity
- sgRNA secondary structures impede formation of active Cas9-sgRNA complexes
- sgRNA and Cas9 concentration influences off-target activity
- Strategies to minimize sgRNA off-target activity
- Conclusion
- References.
- Delivery of CRISPR/Cas9 gene editing components to the cornea
- Summary
- References
- 8 Employing nonhomologous end joining and homology-directed repair for treatment of Leber congenital amaurosis and inherite...
- Leber congenital amaurosis and inherited retinal degeneration
- Genetic aberrations in patients with LCA
- Genetic aberrations in patients with other IRDs
- Genome surgery for treatment of human diseases
- Modes of correction with genome surgery
- Nonhomologous end joining
- Homology-directed repair
- Employing NHEJ and HDR for treatment of LCA and IRD
- Nonhomologous end joining
- Homology-directed repair
- Future direction and conclusions
- References
- 9 The ethics of gene editing in human stem cells
- Overview of legal and political regulation
- Human embryonic stem cells
- Induced pluripotent stem cells
- Germline gene editing
- Looking ahead: the slippery slope
- References
- Index
- Back Cover.