Gene Therapy Science for Health Care Professionals

Welcome to Gene Therapy Science!

Gene Therapy Science is an educational platform intended to further your understanding of the science of gene therapy, with focus on principles specific to hemophilia.

Gene therapy is the introduction, removal or change in genetic material—specifically DNA or RNA—into the cells of a patient to treat a specific disease.¹ In its broadest interpretation, the term “gene therapy” may refer to:

Gene transfer² (gene addition): addition of a functional copy of a missing gene or augmentation of a gene that is non-functional into target cells to produce more of a protein ²

Gene editing:² removal, disruption or correction of faulty elements of DNA within the gene

Cell therapy:² transfer of intact, live cells into a patient

Throughout these webpages, when referring to “gene therapy”, the focus will be on the first of these approaches: gene transfer.

The content on this website will explore areas as they relate to hemophilia, including:

Key Principles

Understand the key principles of hemophilia gene therapy and the science underpinning this approach.

Hemophilia Gene Therapy and the Liver

Review the importance of liver biology in the context of hemophilia gene therapy.

Vector Capsid Design

Explore the building blocks of recombinant adeno-associated virus-derived vectors and the approaches used to optimize the vector capsid for hemophilia gene therapy.

Optimizing Transgene Expression

Learn more about the recombinant adeno-associated virus transgene expression cassette and its role in hemophilia gene therapy. Discover how transgene expression can be optimized.

Gene Therapy and the Immune System

Explore innate and adaptive immune considerations associated with recombinant adeno-associated virus hemophilia gene therapy and methods under investigation to evade such an immune response.

Visit our learning center and news and events page for the latest updates and to explore further resources.