Genetic Hematologic Conditions Resource Library
Explore examples of research, developmental milestones, and investigational treatment approaches targeting the liver.
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Hemophilia Patient Gene Therapy
Gene TherapyDiscover how a desired protein such as FVIII or FIX for hemophilia can be made in a cell by introducing a vector that delivers a new functional gene.
Hemophilia A
HemophiliaLearn how the frequency of bleeding episodes in patients with hemophilia A determines the severity of disease. See how it is diagnosed and managed.
download PDFMilestones in the History of Gene Therapy for Hemophilia
HemophiliaExplore a timeline of important gene therapy milestones related to gene therapies for hemophilia.
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 gene therapies 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.
Liver-Directed AAV Delivery
Gene TherapyWatch how recombinant AAV vectors enter the liver, locate specific surface receptors, and combine with target cells.
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.
Supplemental Resources
Explore additional genetic hematologic conditions resources to supplement conversations with your patients.
Annual Hematology Events
At Spark Therapeutics, we’re striving to address unmet needs in genetic diseases. Here are some events relevant to our research.
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