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CRISPR-Cas-Based Genome Editing for Treating Human Diseases-Part A /

CRISPR-Cas-Based Genome Editing for Treating Human Diseases-Part A, Volume 208 represents CRISPR-Cas systems for genome editing.Currently, CRISPR-Cas systems are proven a key technology for targeted genome editing, which is acting as a simple, rapid, and cost-effective solution.

Bibliographic Details
Main Author: Singh, Vijay, 1950- (Author)
Corporate Author: ScienceDirect (Online service)
Format: eBook
Language:English
Published: London, England : Academic Press, [2024]
Edition:First edition.
Series:Progress in Molecular Biology and Translational Science Series.
Subjects:
Gene therapy.
Genetic Therapy
Thérapie génique.
Electronic books.
Online Access:Connect to the full text of this electronic book
Table of Contents:
  • Front Cover
  • Progress in Molecular Biology and Translational Science
  • Copyright
  • Contents
  • Contributors
  • Preface
  • Chapter One: An overview and potential of CRISPR-Cas systems for genome editing
  • 1 Introduction
  • 2 Zinc finger nucleases (ZFNs)
  • 3 Transcription activator-like effector nucleases (TALENs)
  • 4 CRISPR-Cas systems
  • 5 Evolution of CRISPR-Cas system
  • 6 Mechanism of CRISPR-Cas systems
  • 6.1 Adaptation phase
  • 6.2 Expression and maturation phase
  • 6.3 Interference phase
  • 7 Types of CRISPR-Cas systems
  • 7.1 Type I CRISPR-Cas systems
  • 7.2 Type II CRISPR-Cas systems
  • 7.3 Type III CRISPR-Cas systems
  • 7.4 Type IV CRISPR-Cas systems
  • 7.5 Type V CRISPR-Cas systems
  • 7.6 Type VI CRISPR-Cas systems
  • 8 Applications of CRISPR-Cas systems
  • 8.1 CRISPR-Cas based genome editing
  • 8.2 CRISPR-Cas assisted metabolic pathways engineering
  • 8.3 CRISPR-Cas systems in gene therapy
  • 8.4 CRISPR-Cas systems for removal human viruses
  • 8.5 CRISPR-Cas systems in creating resistance crops
  • 8.6 CRISPR-Cas systems in disease diagnostics
  • 9 Conclusion and future challenges
  • Acknowledgement
  • References
  • Chapter Two: Advances in CRISPR-Cas systems for human bacterial disease
  • 1 Introduction
  • 1.1 Structure and mechanism of the CRISPR-Cas system
  • 1.2 Immune recognition and defense mechanism of CRISPR-Cas system I and II
  • 2 Application of CRISPR-Cas system in human bacterial diseases
  • 2.1 Combating antibiotic resistance
  • 2.1.1 Targeting resistance genes
  • 2.1.2 Plasmid curing
  • 2.2 Targeted bactericidal effects
  • 2.2.1 Direct killing of pathogenic bacteria
  • 2.2.2 Phage-mediated delivery
  • 2.3 Modulating the microbiome
  • 2.3.1 Precision microbiome editing
  • 2.3.2 Preventing horizontal gene transfer (HGT)
  • 2.4 Developing new antimicrobials
  • 2.4.1 Novel antibacterial agents.
  • 2.4.2 Synergistic therapies
  • 2.5 Diagnostic applications
  • 2.5.1 Rapid and accurate diagnostics
  • 2.5.2 Detection of resistance genes
  • 2.6 Vaccines and prophylactics
  • 2.6.1 CRISPR-Cas vaccines
  • 2.6.2 Preventative measures
  • 3 CRISPR-Cas systems to combat antimicrobial resistance
  • 4 CRISPR in precision antibacterials
  • 5 CRISPR in ESKAPE pathogens
  • 5.1 Drug resistance mechanisms
  • 5.1.1 Innate resistance
  • 5.1.2 Acquired resistance
  • 6 CRISPR in tuberculosis
  • 6.1 Historical and epidemiological context of tuberculosis
  • 6.2 Mechanisms and variants of CRISPR-Cas systems
  • 6.3 Advances in CRISPR-Cas systems for tuberculosis diagnostics
  • 6.4 CRISPR in TB treatment and research
  • 6.5 Significance of CRISPR in advancing TB research and treatment
  • 7 CRISPR in pathogen detection
  • 8 Ethical and regulatory considerations
  • 9 Conclusion and future perspectives
  • References
  • Chapter Three: CRISPR-Cas based genome editing for eradication of human viruses
  • 1 Introduction
  • 2 Exploring CRISPR-Cas systems for genome editing of human viruses
  • 2.1 Human immunodeficiency virus
  • 2.2 CRISPR-Cas systems for genome editing of HIV
  • 2.3 HIV-1 resistance through CRISPR-Cas9-mediated CCR5 knockout
  • 2.4 HIV-1 therapy through targeted gene knock-in using SORTS and CRISPR-Cas9 system
  • 3 Hepatitis B virus
  • 3.1 CRISPR-Cas systems to treatment of hepatitis B
  • 3.2 Hepatitis B core and surface antigens
  • 3.3 Application of CRISPR-Cas9 RNPs for HBV elimination
  • 3.4 Targeting HBV RNAs using CRISPR-Cas13b
  • 4 Human papillomavirus
  • 4.1 Gene knockout chain reaction targets (GKCR) in HPV18 E6 and E7
  • 4.2 CRISPR-Cas13a targets HPV E6 gene to combat cancer
  • 5 Severe acute respiratory syndrome-Coronavirus-2 (SARS-CoV-2)
  • 5.1 CRISPR-Cas based detection of SARS-CoV-2
  • 5.2 CRISPR-crafted SARS-CoV-2 vaccine.
  • 6 Conclusion and future remarks
  • Acknowledgments
  • References
  • Chapter Four: Advances in CRISPR-Cas systems for gut microbiome
  • 1 Introduction
  • 2 CRISPR-Cas for probiotic development
  • 2.1 Variety of CRISPR-Cas systems found in probiotics
  • 2.2 Genome engineering in probiotics using CRISPR-Cas systems
  • 2.3 Application of CRISPR-engineered probiotics in therapeutics
  • 2.4 Strategies and challenges for CRISPR based genome editing of probiotics
  • 3 CRISPR-Cas for microbiome diagnostics
  • 3.1 Applications in microbiome diagnostics
  • 3.1.1 Detection of pathogenic bacteria in clinical samples
  • 3.1.2 Identification of antibiotic resistance genes
  • 3.1.3 CRISPR-based diagnostics for microbiome monitoring
  • 3.1.4 Tracking microbiome shifts
  • 3.2 Technological advancements and innovations
  • 3.3 Challenges and limitations
  • 4 CRISPR-Cas for pathogen targeting
  • 5 CRISPR-Cas for microbiome engineering
  • 5.1 Targeting specific microbial genes
  • 5.1.1 Gene knockouts
  • 5.1.2 Gene activation and repression
  • 5.2 Modulating microbial communities
  • 5.2.1 Editing microbial population dynamics
  • 5.2.2 Synthetic biology approaches
  • 5.3 Horizontal gene transfer (HGT) and CRISPR-Cas
  • 5.3.1 Preventing unwanted gene transfer
  • 5.3.2 Harnessing HGT for beneficial traits
  • 6 Ethical and regulatory issues related to use of CRISPR-Cas
  • 7 Conclusion and future perspectives
  • References
  • Chapter Five: Advances in CRISPR-Cas systems for fungal infections
  • 1 Introduction
  • 2 Common methods for changing the genes of fungi
  • 3 Utilization of CRISPR/Cas systems in fungal genetic engineering
  • 3.1 Classification of CRISPR/Cas systems
  • 3.2 The CRISPR/Cas9 system that relies on DNA
  • 3.3 CRISPR/Cas9 ribonucleoproteins (RNPs)
  • 3.4 Utilizing both in vitro and in vivo methods to express the Cas/sgRNA complex.
  • 3.5 Gene editing using CRISPR/Cas12a
  • 3.6 Transcriptional regulation with CRISPR/Cas
  • 3.7 Epigenetic editing using CRISPR/Cas
  • 3.8 Gene editing system utilizing CRISPR/Cas9 technology without the need for genetic markers
  • 4 Current constraints and future potential of CRISPR/Cas-mediated fungi genome engineering
  • 5 Conclusion
  • References
  • Chapter Six: Recent development in CRISPR-Cas systems for human protozoan diseases
  • 1 Introduction
  • 2 Plasmodium
  • 2.1 First developed CRISPR-Cas9 systems for gene editing in P. falciparum
  • 2.2 Enhanced CRISPR-Cas9 systems for gene editing in P. falciparum
  • 2.3 CRISPR-Cas9 based system for tagging endogenous genes of P. falciparum
  • 2.4 CRISPR-Cas9 based system to explore drug resistance in P. falciparum
  • 2.5 CRISPR-Cas9 based conditional knockdown and knockout systems to better characterize essential genes of P. falciparum
  • 2.6 CRISPR-Cas9 based systems to explore epigenetic regulation of essential genes of P. falciparum
  • 2.7 CRISPR-Cas based diagnostic systems
  • 2.8 CRISPR-Cas9 based systems of gene-drive in Anopheles vector for control and elimination of P. falciparum
  • 2.9 CRISPR-Cas9 based generation of transgenic line for assisting in P. falciparum research
  • 2.10 Applying CRISPR-Cas9 technology to enhance the understanding of P. falciparum biology
  • 2.10.1 Study of genes involved in apicoplast biogenesis of P. falciparum
  • 2.10.2 Study of genes involved in drug resistance using CRISPR-Cas9
  • 2.10.3 Study of Anopheles mosquito genes involved in infection by P. falciparum
  • 3 Leishmania
  • 3.1 CRISPR-Cas9 technology in leishmaniasis
  • 3.1.1 Stable CRISPR-Cas9 expression systems
  • 3.1.1.1 U6snRNA promoter for the expression of gRNA
  • 3.1.1.2 RNA polymerase I promoter in the CRISPR-Cas9 system for the expression of sgRNA
  • 3.1.2 Transient CRISPR expression systems.
  • 3.1.2.1 T7 promoter-based system
  • 3.1.2.2 RNP complex of CRISPR-Cas9 system
  • 3.1.2.3 T7-pSP72-based system
  • 3.2 Applications of CRISPR-Cas9 in Leishmania
  • 3.2.1 Desired editing of the target locus for functional analysis and localization studies
  • 3.2.1.1 Incorporation of single point mutations to understand drug resistance mechanism and function of the target gene
  • 3.2.1.2 Generation of gene knockouts
  • 3.2.1.3 Endogenous gene tagging
  • 3.2.2 Targeting multi-gene family and co-selection
  • 3.2.3 Expression of multiple guides using ribozymes
  • 3.2.4 Leishmania diagnostics
  • 3.2.5 CRISPR-Cas9 cytosine base editor (CBE) for large scale screen
  • 3.2.5.1 Gene drive
  • 4 Trypanosoma
  • 4.1 CRISPR-Cas9 systems for gene editing in Trypanosoma
  • 4.1.1 Constitutive Cas9 expression and T7 RNA polymerase-based system
  • 4.1.1.1 Ribonucleoprotein system for gene editing
  • 4.1.1.2 Episome-based CRISPR-Cas9 system
  • 4.1.1.3 Tetracycline-induced Cas9 gene editing in Trypanosoma
  • 4.1.2 Transient CRISPR-Cas9 expression system
  • 4.1.2.1 GFP tagged Cas9 system
  • 4.2 Applications of CRISPR-Cas9 in Trypanosoma
  • 4.2.1 Disruption of multigene family
  • 4.2.2 Functional analysis of Trypanosoma genes
  • 4.2.3 Endogenous tagging of genes to study cellular functions
  • 4.2.4 Improvement in the host-pathogen interaction studies
  • 4.2.5 Conditional regulation of parasite genes
  • 5 Concluding remarks and future aspects
  • Acknowledgements
  • References
  • Chapter Seven: Advances in CRISPR/Cas systems-based cell and gene therapy
  • 1 Introduction
  • 1.1 History and background
  • 1.2 Utility in gene therapy
  • 1.2.1 Precision in genome editing
  • 1.2.2 Functional adaptability
  • 1.2.3 Efficiency of variants
  • 1.3 CRISPR/Cas subtypes: prokaryotic origins and eukaryotic adaptation
  • 2 Delivery formats
  • 2.1 Plasmid systems
  • 2.2 mRNA systems.