Genome Editing for Neurodegenerative Diseases : From Concept to Clinical Trials / edited by Sandeep Kumar Singh [and three others].

Format:

Book

Contributor:

Singh, Sandeep Kumar, editor.

Language:

English

Subjects (All):

Gene editing.

Nervous system--Degeneration--Genetic aspects.

Nervous system.

Physical Description:

1 online resource (262 pages)

Edition:

First edition.

Place of Publication:

London, England : Academic Press, [2025]

Summary:

Genome Editing for Neurodegenerative Diseases: From Concept to Clinical Trials examines recent advances in neurodegenerative disease research and clinical challenges in practical applications.

Contents:

Front Cover

Genome Editing for Neurodegenerative Diseases

Copyright Page

Contents

List of contributors

Introduction

References

1. Therapeutic potential of genome editing tools in neurodegenerative diseases

1.1 Genome editing tools

1.1.1 Zinc-finger nucleases (ZFNs)

1.1.2 Transcription activator-like effector nucleases (TALENs)

1.1.3 Clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9

1.2 Role of genome editing tools in neurodegenerative diseases

1.3 Huntington's disease

1.4 Amyotrophic lateral sclerosis

1.5 Alzheimer's disease

1.6 Parkinson's disease

1.7 Conclusion

2. Genetic and molecular basis of neurodegenerative diseases

2.1 Introduction

2.1.1 Neurodegenerative diseases and associated dysregulations

2.1.2 Neurotransmitter and blood-brain barrier dysregulation

2.1.3 Neurotrophic factors and growth signaling

2.1.4 Synaptic dysfunction and connectivity loss

2.1.5 Metabolic dysregulation in neurodegeneration

2.1.6 Neurodegeneration in nonneuronal cell populations

2.2 Genetic basis of neurodegenerative disorders

2.2.1 Alzheimer disease

2.2.2 Parkinson's disease

2.2.3 Huntington's disease

2.2.4 Amyloid lateral sclerosis

2.3 Molecular basis of neurodegenerative disorders

2.3.1 Oxidative stress

2.3.2 Immune modulation

2.3.3 Abnormal protein accumulation

2.4 Noncoding RNAs in neurodegeneration

2.5 Animal model to study neurodegenerative diseases

2.5.1 The mouse model

2.5.2 Fly model

2.5.3 Zebrafish model

2.6 CRISPR/Cas9 genome editing in disease modeling

2.7 Noninvasive neurobehavioral function tests and multimodel approaches for age-related disease progression

2.7.1 Therapeutics

2.7.1.1 Drug therapies

2.7.1.2 Immunotherapy approaches.

2.7.1.3 Multimodal approaches: targeting multiple pathways simultaneously and patient-centric approaches

2.8 Conclusion

Acknowledgments

3. Genome editing tools: principles, structures, and mechanisms

3.1 Introduction

3.2 Zinc finger nucleases

3.3 DNA-binding domain

3.4 DNA-cleavage domain

3.5 Mechanism of zinc finger nucleases activity

3.5.1 Gene disruption

3.5.2 Gene correction

3.5.3 Gene addition

3.5.4 Creation of enhanced zinc finger nucleases

3.6 Application of zinc finger nucleases in therapy

3.7 Application of zinc finger nucleases in the field of agriculture

3.8 Applications zinc finger nucleases for promotions of animals

3.9 CRISPR/Cas9 genome editing technology

3.10 Commercial application of CRISPR in medical plants

3.10.1 Nicotiana tabacum (L.) (Solanaceae)

3.10.2 Artemisia annua (L.) (Compositae)

3.10.3 Trifolium pratense (L.) (Leguminosae)

3.11 Applications CRISPR the field of livestock

3.12 Transcription activator-like effector nucleases technology

3.12.1 Transcription activator-like effector nucleases specificity

3.12.2 Transcription activator-like effector nucleases synthesis

3.12.3 Ligation based on golden gate cloning

3.12.4 PCR-based ligation via golden gate

3.12.5 Restriction enzymes assembly ligation

3.12.6 Fast ligation-based automatable solid-phase high-throughput system

3.13 Mechanism of transcription activator-like effector nuclease activity

3.14 Biomedical applications of transcription activator-like effector nuclease

3.15 Conclusions and future perspective

4. Advances in CRISPER/Cas system and genome editing technologies for the treatment of neurodegenerative diseases

4.1 Introduction

4.2 Gene-editing tools

4.2.1 Homologous recombination

4.2.2 Zinc finger nucleases.

4.2.3 Transcription activator-like effector nuclease

4.2.4 Clustered regularly interspaced palindromic repeat

4.3 Recent advances in the genomes editing

4.4 Therapeutic approaches of neurodegenerative diseases using genome editing technologies

4.4.1 Alzheimer's diseases

4.4.2 Parkinson's disease

4.4.3 Huntington's disease

4.4.4 Amyotrophic lateral sclerosis

4.5 Concluding remarks and future prospective

5. Use of CRISPR system in genetic screening to detect neurodegenerative disease

5.1 Introduction

5.2 A brief summary of CRISPR

5.2.1 Process involved in the perturbation of genes

5.2.2 Editing of genome

5.2.3 Gene expression control

5.2.4 Aiming mRNAs

5.2.5 Screening of CRISPR in mammalian cells

5.2.5.1 Genetic screening by pooling

5.2.5.2 Genetic screening by array

5.2.5.3 Systematic genetic screening

5.2.5.4 Screening in induced pluripotent stem cell-derived cells

5.3 Role of CRISPR in modeling of disease

5.3.1 Mechanism of variants of the disease

5.3.2 Therapeutic targets discovery

5.3.3 Alteration in expression of gene

5.4 Implementation of CRISPR in neurodegenerative disease

5.4.1 Alzheimer's disease

5.4.2 Parkinson's disease

5.4.3 Huntington's disease

5.4.4 Amyotrophic lateral sclerosis

5.5 Challenges and limitations

5.6 Future directions

5.7 Conclusion

6. Epigenetic manipulation and gene regulation using the CRISPR/Cas system for neurodegenerative diseases

6.1 Introduction

6.2 What is epigenetic modification?

6.3 History of epigenetics

6.3.1 Brief overview of early concepts

6.3.2 Impact of epigenetic functions in health and disease

6.4 Types of epigenetic modifications

6.4.1 DNA methylation

6.4.2 Histone modifications

6.4.3 Noncoding RNA interactions (microRNAs).

6.5 CRISPR/Cas9 mediated epigenetics and modifications in neurodegenerative diseases (NDDs)

6.5.1 Brief overview

6.5.2 CRISPR/Cas9 applications as a gene editing tool in neuroscience

6.6 Limitations and conclusions

7. Transgene delivery system: viral, nonviral, and other methods for central nervous system

7.1 Introduction

7.2 Viral vectors

7.2.1 Adeno-associated vectors

7.2.2 Lentiviruses

7.2.3 Herpes simplex virus

7.2.4 Retrovirus vectors

7.2.5 Pox virus

7.2.6 Epstein-Barr virus

7.3 Nonviral vectors

7.3.1 Cationic polymer

7.3.2 Cationic lipids

7.3.3 Nanoparticles as nanocarriers

7.3.4 Naked DNA

7.4 Other emerging methods

7.4.1 Exosomes

7.4.2 Electroporation

7.4.3 Ultrasound

7.4.4 Sonodynamics delivery

7.4.5 Magnetic nanoparticles

7.4.6 Cell-based delivery

7.4.7 Ex vivo gene therapy

7.5 Conclusion

8. Gene therapies for neurodegenerative disease: current knowledge and challenges

8.1 Introduction

8.2 Neurodegenerative disease

8.2.1 Alzheimer's disease

8.2.2 Parkinson's disease

8.2.3 Huntington's disease

8.3 Gene therapy for neurodegenerative diseases

8.3.1 Canonical DNA editing

8.3.2 Noncanonical DNA editing

8.3.3 RNA editing

8.3.4 Gene expression-based editing

8.3.5 DNA editing

8.3.6 CRISPR-Cas9

8.3.7 RNA editing

8.3.8 Antisense oligonucleotides

8.4 Challenges in gene therapy

8.4.1 Crossing the blood-brain barrier with adeno-associated vectors

8.4.2 Adenovirus-associated virus based

8.5 Conclusion

9. Gene therapy under clinical trials for neurodegenerative diseases

9.1 Introduction

9.2 Gene therapy

9.3 Preclinical studies to clinical trials

9.3.1 Parkinson's disease

9.3.1.1 AAV2-GAD

9.3.1.2 AAV2-AADC

9.3.1.3 GDNF

9.3.2 Huntington's disease.

9.3.3 Alzheimer's disease

9.3.4 Amyotrophic lateral sclerosis

9.3.5 Spinal muscular atrophy

9.4 Clinical challenges

9.5 Concluding remarks and future prospects

10. Clinical challenges and future prospective in use of gene-editing tools as neurotherapeutics

10.1 Introduction

10.2 Gene therapies as neurotherapeutics

10.2.1 Effects of gene therapies on aging and neurodegenerative diseases

10.2.2 The promise of gene editing in treating neurological disorders

10.2.3 Purpose and scope of gene-editing tools as neurotherapeutics

10.3 Clinical development of neurotherapeutics

10.3.1 Clinical development of gene therapy with approved products

10.3.2 Completed clinical trials on available neurotherapeutics

10.3.3 Progress in early clinical development of promising approaches

10.3.4 The tools used

10.3.5 The CRISPR-Cas approaches

10.3.6 The vectors used

10.4 Prevalent neurodegenerative diseases and gene editing

10.5 Conclusion and outlook on clinical development of gene therapies

Index

Back Cover.

Notes:

Includes bibliographical references and index.

Description based on publisher supplied metadata and other sources.

Description based on print version record.

ISBN:

9780443238260

044323826X

OCLC:

1465668572