This introductory video explains how genes and DNA function and how genetic changes cause hemophilia A and B. It provides an overview of three genetic technologies currently being studied or used in hemophilia care: AAV gene therapy, lentiviral gene therapy, and gene editing. This is the first video in a four-part series on genetic technologies for hemophilia.

This video provides an overview of AAV gene therapy for hemophilia, including how it works, who may be eligible, its potential benefits and limitations, and follow-up considerations. This is the second video in a four-part series on genetic technologies for hemophilia.

This video explains lentiviral gene therapy, an emerging treatment approach being studied for hemophilia A. It describes 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. The video also explores potential benefits, safety considerations, and the current status of research and clinical development. This is the third video in a four-part series on genetic technologies for hemophilia.

This video introduces gene editing as a promising investigational treatment for hemophilia A and B. It explains how CRISPR-based technologies can precisely target DNA to insert therapeutic genes, reviews the different delivery approaches currently being studied, and discusses the potential advantages and remaining unanswered questions surrounding this rapidly evolving field. This is the fourth video in a four-part series on genetic technologies for hemophilia.