Educational Resources
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.
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AAV Gene Therapy Research Animation
Gene TherapyAdeno-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?
Gene TherapyProteins 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
Gene TherapyLearn how genetic therapy approaches can potentially correct genetic mutations to provide functional protein.
Biological Features of a Recombinant AAV
Gene TherapyAAVs 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
Gene TherapyWatch how recombinant AAV vectors delivered into the body locate specific surface receptors of target cells.
Delivery into Target Cells
Gene TherapyDNA 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
Gene TherapyAdministering 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
Gene TherapyThis 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.
download PDFMilestones in the History of Gene Therapy
Gene TherapyExplore a timeline of important gene therapy milestones related to therapies for rare genetic diseases.
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