Resources to educate on gene therapy research
Gene Transfer 101
A simple one page diagram helping patients and caregivers to understand the 5 steps involved in gene transfer research.
Getting to Know Gene Therapy Research:
Behind the Science and Practical Considerations
Annette von Drygalski, MD, PharmD, RMSK
Director, Hemophilia and Thrombosis Treatment Center at University of California San Diego Health
Professor of Clinical Medicine, UC San Diego, California
A background on gene therapy research
With 2,500 studies completed, ongoing, or initiated worldwide, gene therapy is being evaluated to determine the benefits and risks for a range of genetic conditions, including hemophilia A and B. Some gene therapies have already been approved for treatment of certain conditions, including a genetic eye disease and spinal muscular atrophy. No gene therapies for hemophilia A or B have been approved for use or determined to be safe or effective.
How is gene transfer designed to work?
In gene transfer research, a working copy of a mutated gene is designed in a laboratory to provide instructions for the body to make the proteins it needs. That working gene is placed in a protective viral shell and delivered through one-time infusion. Once inside the body, the functional gene is designed to provide instructions for the body to produce the missing or mutated gene. Ongoing research monitors clinical trial participants to gather data to better understand short as well as long term safety and efficacy.