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Introduction

The use of CRISPR-based gene editing technology has revolutionized the field of genetic engineering, offering unprecedented precision, efficiency, and versatility in the modification of DNA sequences. One area where CRISPR has shown great promise is in the treatment of genetic disorders, such as Friedreichʼs ataxia, a rare neurodegenerative disease characterized by progressive loss of coordination and muscle strength. In this thesis, we aim to assess the potential of CRISPR-based gene editing as a therapeutic strategy for Friedreichʼs ataxia.

Chapter 1
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 Friedreich’s ataxia
2.2 Genetic basis of Friedreich’s ataxia
2.3 Current treatment strategies
2.4 CRISPR-based gene editing technology
2.5 Applications of CRISPR in genetic disorders
2.6 CRISPR in neurodegenerative diseases
2.7 Studies on CRISPR-based treatment for Friedreich’s ataxia
2.8 Challenges and ethical considerations of CRISPR technology
2.9 Future directions in CRISPR research
2.10 Summary of literature review

Chapter 3 – Research Methodology
3.1 Research design
3.2 Selection of target gene for editing
3.3 CRISPR/Cas9 delivery methods
3.4 In vitro and in vivo studies
3.5 Data analysis techniques
3.6 Ethical considerations
3.7 Budget and timeline
3.8 Limitations of the methodology

Chapter 4 – Discussion of Findings
4.1 Efficacy of CRISPR-based gene editing in treating Friedreich’s ataxia
4.2 Safety concerns and potential off-target effects
4.3 Comparison of CRISPR with other gene editing technologies
4.4 Future implications and challenges
4.5 Recommendations for further research
4.6 Conclusion

Chapter 5 – Conclusion and Summary
5.1 Summary of key findings
5.2 Implications for the field
5.3 Limitations of the study
5.4 Future research directions
5.5 Conclusion

Thesis Overview:

Friedreich’s ataxia is a devastating neurodegenerative disorder for which there is currently no cure. Recent advances in CRISPR-based gene editing technology have offered new hope for the development of targeted therapies for genetic diseases. This thesis aims to assess the potential of CRISPR-based gene editing for the treatment of Friedreich’s ataxia.

The literature review will provide a comprehensive overview of Friedreich’s ataxia, the genetic basis of the disease, current treatment strategies, and the application of CRISPR technology in genetic disorders and neurodegenerative diseases. The research methodology will outline the design of in vitro and in vivo studies, the selection of target genes for editing, delivery methods for CRISPR/Cas9 components, data analysis techniques, ethical considerations, and budget constraints.

The discussion of findings will analyze the efficacy of CRISPR-based gene editing in treating Friedreich’s ataxia, potential safety concerns and off-target effects, comparison with other gene editing technologies, future implications, and recommendations for further research. The conclusion and summary chapter will summarize key findings, discuss implications for the field, identify limitations of the study, suggest future research directions, and provide a conclusive statement on the potential of CRISPR-based gene editing for the treatment of Friedreich’s ataxia.

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