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Introduction

Alternative splicing is a crucial mechanism that allows a single gene to code for multiple protein variants. This process, regulated by splicing factors, is essential for cellular function and development. Dysregulation of alternative splicing has been implicated in various diseases, including cancer, neurological disorders, and genetic syndromes. In recent years, there has been growing interest in pharmacologically targeting alternative splicing as a therapeutic strategy.

This thesis aims to provide a comprehensive overview of pharmacological modulation of alternative splicing. Specifically, it will explore the potential of small molecules, antisense oligonucleotides, and RNA-targeting therapies in modulating splicing events. The thesis will also discuss the challenges and opportunities in this emerging field and highlight the significance of understanding alternative splicing in drug development.

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 Alternative Splicing
2.2 Regulation of Alternative Splicing
2.3 Role of Alternative Splicing in Disease
2.4 Pharmacological Modulation of Alternative Splicing
2.5 Small Molecule Inhibitors and Activators
2.6 Antisense Oligonucleotides
2.7 RNA-targeting Therapies
2.8 Challenges in Pharmacological Modulation of Alternative Splicing
2.9 Opportunities for Drug Development
2.10 Future Directions

Chapter 3: Research Methodology
3.1 Research Design
3.2 Data Collection Methods
3.3 Sample Selection
3.4 Data Analysis Techniques
3.5 Experimental Approaches
3.6 In vitro and In vivo Models
3.7 Pharmacological Screening Assays
3.8 Bioinformatics Analysis

Chapter 4: Discussion of Findings
4.1 Pharmacological Modulation of Splicing Factors
4.2 Effects of Small Molecule Inhibitors and Activators
4.3 Antisense Oligonucleotide Therapies
4.4 RNA-targeting Therapies in Disease Models
4.5 Mechanisms of Action
4.6 Efficacy and Toxicity Profiles
4.7 Comparative Analysis of Different Approaches
4.8 Challenges and Future Directions

Chapter 5: Conclusion and Summary
5.1 Summary of Findings
5.2 Implications for Drug Development
5.3 Future Perspectives
5.4 Conclusion

Thesis Overview:

Pharmacological modulation of alternative splicing is a promising strategy for developing new treatments for a range of diseases. This thesis will provide a comprehensive overview of the current state of research in this field, focusing on the potential of small molecules, antisense oligonucleotides, and RNA-targeting therapies. The literature review will explore the role of alternative splicing in disease, the mechanisms of splicing regulation, and the challenges and opportunities in pharmacological modulation.

The research methodology section will outline the experimental approaches and data analysis techniques used in the study. The discussion of findings will present the results of pharmacological modulation of splicing factors and the effects of different therapeutic approaches in disease models. The conclusion will summarize the key findings, discuss the implications for drug development, and suggest future directions for research in this field.

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