The video series provides an overview of the science behind hemophilia and explores three genetic technologies currently being used or studied: adeno-associated virus (AAV) gene therapy, lentiviral gene therapy, and gene editing. Using straightforward explanations and visual analogies, the videos explain how these approaches work, their potential benefits, and important considerations.
Genetic Technologies: The Basics
Learn how genes and DNA work, how hemophilia is caused, and the key differences between AAV gene therapy, lentiviral gene therapy, and gene editing.
Genetic Technologies: AAV Gene Therapy
An overview of AAV gene therapy for hemophilia, including how it works, who may be eligible, and its potential benefits and limitations.
Genetic Technologies: Lentiviral Gene Therapy
Discover how lentiviral gene therapy uses a person’s own blood stem cells to introduce a functional clotting factor gene and support long-term clotting factor production.
Genetic Technologies: Gene Editing
Learn how gene editing technologies such as CRISPR can precisely modify DNA and their potential as future treatments for hemophilia.
The videos are available in English, with French and Spanish subtitles.
The four-part educational video series on genetic technologies for hemophilia was exclusively supported by Regeneron.