Explore educational resources related to rare genetic diseases and investigational gene therapy approaches.
Gene Therapy Through the Years
Gene therapy research has advanced over the past 50 years—bringing hope to millions of people living with rare genetic diseases. To learn more about the progress in this field, browse the content below.
AAV Gene Therapy Research Animation
Adeno-associated viral (AAV) vectors are designed to encapsulate genetic material for the potential treatment of genetic diseases. Explore the science behind AAV gene therapy research.
What Causes Genetic Disease?
Proteins carry out a variety of cellular and physiological functions in the body. See how changes in a person’s DNA can affect proteins and lead to genetic disease.
Gene Therapy Strategies
Learn how genetic therapy approaches can potentially correct genetic mutations to provide functional protein.
Biological Features of a Recombinant AAV
AAVs are commonly used as vectors in gene therapy. See how recombinant AAV vectors are designed and how they function in the body.
Targeted Cell AAV Delivery
Watch how recombinant AAV vectors delivered into the body locate specific surface receptors of target cells.
Delivery into Target Cells
DNA is delivered to the nucleus of the target cell to allow for gene expression of the relevant protein. Watch the process in action.
Gene Therapy and the Immune Response
Administering AAV vectors can activate different branches of the immune system. Learn about this process and research to develop immunosuppressive techniques.
A Pathway to Turning Genes into Medicine
This educational experience provides a unique perspective into our approach to gene therapy research. Click through the steps and watch them animate in high-resolution augmented reality.
Milestones in the History of Gene Therapy
Explore a timeline of important gene therapy milestones related to therapies for rare genetic diseases.
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