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Introduction

Huntingtonʼs disease (HD) is a debilitating neurodegenerative disorder that currently lacks a cure. However, recent advancements in gene editing technology, particularly CRISPR-based gene editing, have provided new hope for potential treatments. CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) has revolutionized the field of molecular biology by offering a precise and efficient tool for editing the genetic code of organisms. This thesis aims to investigate the potential of CRISPR-based gene editing 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 treatment options for Huntingtonʼs disease
2.4 CRISPR-based gene editing technology
2.5 Applications of CRISPR in other genetic disorders
2.6 Previous studies on CRISPR for Huntingtonʼs disease
2.7 Ethical considerations of gene editing for human diseases
2.8 Challenges in implementing CRISPR for Huntingtonʼs disease
2.9 Future directions in CRISPR research for Huntingtonʼs disease

Chapter 3: Research Methodology
3.1 Research design
3.2 Sample selection
3.3 Data collection methods
3.4 CRISPR gene editing techniques
3.5 Cell culture and animal models
3.6 Data analysis
3.7 Ethical considerations
3.8 Timeline for the study

Chapter 4: Discussion of Findings
4.1 Efficacy of CRISPR-based gene editing in Huntingtonʼs disease
4.2 Safety considerations of CRISPR therapy
4.3 Potential challenges and limitations
4.4 Comparison with other treatment options
4.5 Future implications for clinical use
4.6 Ethical considerations in gene editing research
4.7 Regulatory approval process
4.8 Cost-benefit analysis

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

Thesis Overview

Huntingtonʼs disease (HD) is a devastating neurological disorder caused by a mutation in the HTT gene, leading to progressive loss of motor control, cognition, and emotional stability. Currently, there is no cure for HD, and available treatments only aim to manage symptoms. However, with the emergence of CRISPR-based gene editing technology, there is newfound hope for targeted and precise treatment of genetic disorders like HD.

This thesis aims to investigate the potential of CRISPR-based gene editing for the treatment of Huntingtonʼs disease. The literature review will provide an overview of HD, the genetics of the disease, current treatment options, and the fundamentals of CRISPR technology. Previous studies on CRISPR for HD, ethical considerations, and future directions in research will also be explored.

The research methodology will outline the design of the study, sample selection, data collection methods, CRISPR gene editing techniques, and ethical considerations. Cell culture and animal models will be utilized to assess the efficacy and safety of CRISPR therapy for HD.

The discussion of findings will analyze the efficacy of CRISPR-based gene editing in HD, safety considerations, potential challenges, and future implications for clinical use. Ethical considerations, regulatory approval processes, and cost-benefit analyses will also be addressed.

In conclusion, this thesis aims to provide a comprehensive overview of the potential of CRISPR-based gene editing for the treatment of Huntingtonʼs disease. By exploring the current state of research, ethical considerations, and future directions, this study hopes to contribute to the advancement of novel therapies for HD patients.

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