Description

This course presents an introduction to cell and gene therapy. It covers traditional non-genetic cell therapies, in-vivo gene therapy, and ex-vivo gene therapy. Basic genetics and lab techniques are covered, along with an overview of AAV manufacturing for gene delivery and FDA requirements for approval.

Course Objectives

• Explain the difference between cell culture, cell therapy, and gene therapy.
• Distinguish between non-genetic and genetic cell therapy (ex-vivo).
• Describe the principles of genetics and its importance in understanding therapies.
• Identify key analytical equipment used in gene therapy.
• Summarize the AAV manufacturing process.
• Understand FDA approval process and long-term concerns.

Outline of Instruction

1. Introduction: Comparison of cell culture, cell therapy, in-vivo and ex-vivo gene therapy
2. Review of Fundamentals: Cells, DNA/RNA, genes, mutations
3. Key Analytical Technologies: Protein assays, amplification, sequencing
4. Cell Therapy: Blood transfusion, stem cell, immune cell therapy
5. Gene Therapy: Targets, delivery techniques, editing, in-vivo & ex-vivo
6. AAV Manufacturing Process
7. Regulations & FDA Guidance