Investigating the potential of CRISPR-based gene editing for the treatment of Duchenne muscular dystrophy – Complete Phd and Masters Thesis

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Introduction

Duchenne muscular dystrophy (DMD) is a severe and progressive muscle-wasting disease that affects approximately 1 in 5000 male births worldwide. This genetic disorder is caused by mutations in the dystrophin gene, leading to the absence of the dystrophin protein in muscle cells. Despite advances in our understanding of DMD, there is currently no cure for this devastating condition. However, recent advancements in gene editing technologies, particularly CRISPR-based gene editing, have opened up new possibilities for the treatment of genetic disorders like DMD.

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 Genetics of Duchenne muscular dystrophy
2.2 Current treatment options for DMD
2.3 Overview of CRISPR-based gene editing
2.4 Applications of CRISPR in gene therapy
2.5 CRISPR as a potential treatment for DMD
2.6 Challenges and limitations of CRISPR technology
2.7 Ethical considerations of gene editing for DMD
2.8 Regulatory framework for gene editing in medical research
2.9 Recent advances in CRISPR-based gene editing for DMD
2.10 Future directions in CRISPR research for DMD

Chapter 3: Research Methodology
3.1 Research design and approach
3.2 Selection of study participants
3.3 Data collection methods
3.4 Data analysis techniques
3.5 Experimental procedures for CRISPR-based gene editing
3.6 Evaluation of treatment outcomes
3.7 Ethical considerations in research
3.8 Validation of research findings

Chapter 4: Discussion of Findings
4.1 Efficacy of CRISPR-based gene editing in DMD treatment
4.2 Safety and potential risks of CRISPR therapy
4.3 Long-term effects of gene editing on patients with DMD
4.4 Comparison of CRISPR therapy with traditional treatment options
4.5 Challenges and limitations of implementing CRISPR therapy in clinical settings
4.6 Ethical considerations in the use of CRISPR technology
4.7 Regulatory challenges and guidelines for gene editing research
4.8 Future implications and directions for CRISPR research in DMD therapy

Chapter 5: Conclusion and Summary
In conclusion, this thesis aims to investigate the potential of CRISPR-based gene editing for the treatment of Duchenne muscular dystrophy. By reviewing the current literature, evaluating research methodology, discussing findings, and summarizing the implications of this research, we hope to contribute to the ongoing efforts to find a cure for DMD using innovative gene editing technologies. The findings of this study may provide valuable insights for researchers, clinicians, and policymakers involved in the development and implementation of CRISPR therapy for DMD.

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