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Introduction

Duchenne muscular dystrophy (DMD) is a devastating genetic disorder characterized by progressive muscle degeneration and weakness, ultimately leading to loss of ambulation and premature death in affected individuals. Although there is currently no cure for DMD, recent advancements in gene therapy, particularly CRISPR-based gene editing technology, have shown promising results in preclinical studies for treating this debilitating disease. This thesis aims to investigate the potential of CRISPR-based gene therapy for the treatment of Duchenne muscular dystrophy.

1.1 Introduction
1.2 Background of the study
1.3 Problem Statement
1.4 Objectives of the study
1.5 Limitations of the study
1.6 Scope of the study
1.7 Significance of the study
1.8 Structure of the Thesis
1.9 Definition of Terms

Chapter 2: Literature Review
2.1 Overview of Duchenne muscular dystrophy
2.2 Current treatment options for DMD
2.3 CRISPR-based gene editing technology
2.4 Previous studies on CRISPR-based gene therapy for DMD
2.5 Challenges and limitations of CRISPR-based gene therapy
2.6 Ethical considerations in gene editing for genetic disorders
2.7 Regulatory framework for gene therapy research
2.8 Future prospects of CRISPR-based gene therapy for DMD
2.9 Comparison of CRISPR with other gene editing technologies
2.10 Potential risks and concerns associated with CRISPR-based gene therapy

Chapter 3: Research Methodology
3.1 Research design
3.2 Study population
3.3 Sampling techniques
3.4 Data collection methods
3.5 Data analysis procedures
3.6 Ethical considerations
3.7 Pilot study
3.8 Validity and reliability of data

Chapter 4: Discussion of Findings
4.1 Overview of study findings
4.2 Analysis of data collected
4.3 Comparison with existing literature
4.4 Implications for future research
4.5 Recommendations for clinical practice
4.6 Limitations of the study
4.7 Strengths of the study
4.8 Areas for further research

Chapter 5: Conclusion and Summary
5.1 Summary of key findings
5.2 Contribution to existing knowledge
5.3 Practical implications
5.4 Limitations of the study
5.5 Recommendations for future research
5.6 Conclusion

Thesis Overview

Duchenne muscular dystrophy (DMD) is a severe and progressive genetic disorder that affects approximately 1 in every 3,500 male births worldwide. It is caused by mutations in the dystrophin gene, leading to the absence of the dystrophin protein in muscle cells. Currently, there is no cure for DMD, and treatment options are limited to supportive care and management of symptoms to improve quality of life for affected individuals.

Recent advancements in gene therapy have provided new hope for the treatment of genetic disorders such as DMD. CRISPR-based gene editing technology, in particular, has shown great potential for correcting disease-causing mutations in the genome and restoring normal gene function. This thesis aims to investigate the potential of CRISPR-based gene therapy for the treatment of Duchenne muscular dystrophy, with a focus on addressing the following key objectives:

1. To review the current literature on Duchenne muscular dystrophy, including the pathophysiology of the disease and existing treatment options.
2. To evaluate the advantages and limitations of CRISPR-based gene editing technology for correcting dystrophin gene mutations in DMD.
3. To analyze previous studies on CRISPR-based gene therapy for DMD, including preclinical and clinical trials.
4. To assess the ethical considerations, regulatory framework, and potential risks associated with using CRISPR for gene therapy in genetic disorders.
5. To explore the future prospects of CRISPR-based gene therapy for DMD and compare it with other gene editing technologies.
6. To discuss the implications of the study findings for clinical practice, research, and the development of novel therapeutic interventions for DMD.

Through a comprehensive literature review, detailed research methodology, and in-depth discussion of findings, this thesis will provide valuable insights into the potential of CRISPR-based gene therapy for the treatment of Duchenne muscular dystrophy. The findings of this study are expected to contribute to the advancement of gene therapy research and the development of innovative treatments for DMD and other genetic disorders.

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