About the course
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 Stanford Genetics and Genomics Certificate.
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
Time to Complete
You should expect to spend 10-18 hours to complete each course, depending on your familiarity with the topic.
For individual courses, we recommend that you designate 2-3 hours per week to watch video lectures and complete assignments in order to finish within 60 days.
Since the All-Access Plan allows you to access all courses for one year, you can determine how quickly to progress, but you must complete courses with 365 days in order to receive credit.
You can earn the Stanford Genetics and Genomics Certificate by successfully completing the two required and any four elective courses. You may enroll in courses individually or through the All-Access Plan.
Administrative Appointments Founder of LogicBio Therapeutics, a gene therapy company (2014) Member of the American Society of Gene and Cell therapy (2011) Honors & Awards Presidential symposium lecturer at the annual meeting of the American Society for Gene and Cell Therapy (ASGCT) (2014) ...
Mark A. Kay, MD, PhD, is the Director of the Program in Human Gene Therapy, and Professor in the Department of Pediatrics and Genetics at Stanford University School of Medicine. Dr. Kay is one of the founders of the American Society of Gene Therapy and served as its President in 2005-2006. Dr. Ka...
Maria Grazia Roncarolo, MD is the co-director of the Institute for Stem Cell Biology and Regenerative Medicine, the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine (blood and marrow transplantation), chief of the Division of Pediatric Stem...
Videos and materials
Because of COVID-19, many providers are cancelling or postponing in-person programs or providing online participation options.
We are happy to help you find a suitable online alternative.