Investigating the potential of CRISPR-based gene therapy for the treatment of Huntingtonʼs disease – Complete Phd and Masters Thesis

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Introduction

Huntingtonʼs disease (HD) is a devastating neurodegenerative disorder characterized by progressive motor dysfunction, cognitive decline, and psychiatric disturbances. Currently, there is no cure for HD, and existing treatments only manage symptoms without addressing the underlying cause of the disease. However, recent advancements in gene therapy, particularly CRISPR-based gene editing technology, have sparked hope for a potential cure for HD. This thesis aims to investigate the potential of CRISPR-based gene therapy for the treatment of Huntingtonʼs disease.

Chapter 1: Introduction
1.1 Introduction
1.2 Background of study
1.3 Problem Statement
1.4 Objective of study
1.5 Limitation of study
1.6 Scope of study
1.7 Significance of study
1.8 Structure of the Thesis
1.9 Definition of terms

Chapter 2: Literature Review
2.1 Overview of Huntingtonʼs disease
2.2 Genetics of Huntingtonʼs disease
2.3 Current treatments for Huntingtonʼs disease
2.4 CRISPR-based gene therapy
2.5 Applications of CRISPR in treating genetic disorders
2.6 Case studies of CRISPR-based gene therapy in other neurological disorders
2.7 Ethical considerations of gene editing in humans
2.8 Challenges and limitations of CRISPR-based gene therapy
2.9 Future directions in gene therapy for Huntingtonʼs disease
2.10 Summary of literature review

Chapter 3: Research Methodology
3.1 Research design
3.2 Sample selection
3.3 Data collection methods
3.4 Data analysis techniques
3.5 Ethical considerations
3.6 Timeline of the study
3.7 Budget allocation
3.8 Potential risks and mitigation strategies

Chapter 4: Discussion of Findings
4.1 Analysis of data
4.2 Comparison of findings with existing literature
4.3 Implications of the results
4.4 Recommendations for future research
4.5 Practical applications of CRISPR-based gene therapy for Huntingtonʼs disease
4.6 Limitations of the study
4.7 Strengths of the study
4.8 Conclusion

Chapter 5: Conclusion and Summary
5.1 Summary of key findings
5.2 Contributions to the field
5.3 Implications for clinical practice
5.4 Limitations of the study
5.5 Recommendations for future research
5.6 Concluding remarks

Thesis Overview:

Investigating the potential of CRISPR-based gene therapy for the treatment of Huntingtonʼs disease is a comprehensive study that aims to explore the application of cutting-edge gene editing technology in the context of a debilitating neurodegenerative disorder. The thesis begins with a detailed introduction that sets the stage for the investigation, providing background information on Huntingtonʼs disease, outlining the problem statement, objectives, limitations, scope, significance of the study, and defining key terms.

The literature review in Chapter 2 offers an extensive analysis of existing research on HD, gene therapy, CRISPR technology, ethical considerations, challenges, and future directions in the field. This section provides a solid foundation for the research methodology, which is detailed in Chapter 3. The research methodology outlines the design, sample selection, data collection, analysis techniques, ethical considerations, timeline, budget, and potential risks of the study.

Chapter 4 delves into the discussion of findings, analyzing the data collected, comparing it with existing literature, and drawing implications for clinical practice. The chapter also offers recommendations for future research and practical applications of CRISPR-based gene therapy for HD.

Finally, Chapter 5 presents a conclusion and summary of the thesis, summarizing key findings, highlighting contributions to the field, discussing implications for clinical practice, outlining limitations of the study, and offering recommendations for future research. This thesis aims to contribute to the growing body of knowledge on gene therapy for Huntingtonʼs disease and inform future research in the field.

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