1. Introduction
1.1 Background and significance
1.2 Objectives of the study
2. Biochemical Principles of Gene Delivery
2.1 Viral vectors and their biochemical properties
2.2 Non-viral vectors and gene delivery mechanisms
2.3 Cellular uptake and intracellular trafficking of genes
3. Gene Expression and Regulation
3.1 Transcriptional control and promoter elements
3.2 Post-transcriptional modifications and mRNA stability
3.3 Epigenetic regulation and gene silencing
4. Biochemical Consequences of Gene Therapy
4.1 Immune responses and host reactions
4.2 Integration and stability of therapeutic genes
4.3 Off-target effects and genotoxicity
5. Gene Editing Technologies
5.1 CRISPR-Cas9 and targeted genome editing
5.2 Zinc finger nucleases and TALENs
5.3 Biochemical mechanisms of gene editing
This project aims to analyze the role of biochemistry in gene therapy. Gene therapy holds great promise for treating genetic disorders, cancer, and other diseases by introducing therapeutic genes into target cells. Understanding the biochemical processes involved in gene delivery, gene expression, and gene regulation is essential for the development of safe and effective gene therapy strategies. This project will involve studying the biochemistry of viral and non-viral gene delivery systems, gene expression vectors, and gene editing technologies. The focus will be on the mechanisms of gene transfer, gene regulation, and the biochemical consequences of gene therapy. The findings from this research will contribute to the advancement of gene therapy approaches and the translation of gene-based treatments into clinical practice.
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