Principles and Practices of Gene Therapy

Description

Gene therapy is promising to be key in the battle against cancer, inherited disorders, and many other diseases. Decades worth of advances in this field have resulted in a growing number of successful clinical trials to develop safe and effective treatments. Over the past few years scientists have developed a number of new nucleic acid-based therapies, which continue to improve the versatility of these genetic-based treatment approaches. In this course, you will start by building a fundamental understanding of gene therapy, then dive deeper with an in-depth look at important trends, research and advances in gene therapy. You will gain a clear understanding of how gene therapy works, how it has developed and advanced, and how much potential it has.

This course is an elective course in the Genetics and Genomics Program.

What you will learn

• Basic principles for getting nucleic acids into cells and using viruses to transfer DNA
• Proven and new approaches to clinical trials
• How to effectively use genome editing tools
• Methodologies for successful RNAi and expression of non-coding RNAs to regulate genes and treat disease