Hematologic & Lysosomal Storage Disorders Resource Library

Explore examples of research, developmental milestones, and investigational treatment approaches targeting the liver.


Explore the Science of Gene Therapy Research Preview

Explore the Science of Gene Therapy Research


Understand the science behind gene therapy and learn how it could help address rare genetic diseases like late-onset Pompe disease.

Hemophilia A Preview

Hemophilia A


Learn how the frequency of bleeding episodes in patients with hemophilia A determines the severity of disease. See how it is diagnosed and managed.

Pompe Disease Preview

Pompe Disease


Review clinical characteristics, diagnosis and testing options, management, and more for Pompe disease.

Milestones in the History of Gene Therapy for Hemophilia


Explore a timeline of important gene therapy milestones related to gene therapies for hemophilia.

AAV Gene Therapy Research Animation Preview

AAV Gene Therapy Research Animation

Gene Therapy

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? Preview

What Causes Genetic Disease?

Gene Therapy

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 Preview

Gene Therapy Strategies

Gene Therapy

Learn how genetic therapy approaches can potentially correct genetic mutations to provide functional protein.

Biological Features of a Recombinant AAV Preview

Biological Features of a Recombinant AAV

Gene Therapy

AAVs 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 Preview

Liver-Directed AAV Delivery

Gene Therapy

Watch how recombinant AAV vectors enter the liver, locate specific surface receptors, and combine with target cells.

Delivery into Target Cells Preview

Delivery into Target Cells

Gene Therapy

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 Preview

Gene Therapy and the Immune Response

Gene Therapy

Administering AAV vectors can activate different branches of the immune system. Learn about this process and research to develop immunosuppressive techniques.

Supplemental Resources

Explore additional hematologic and lysosomal storage disorder resources to supplement conversations with your patients.

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