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.


Gene Therapy
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.

Targeted Cell AAV Delivery Preview

Targeted Cell AAV Delivery

Gene Therapy

Watch how recombinant AAV vectors delivered into the body locate specific surface receptors of 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.

A Pathway to Turning Genes into Medicine Preview

A Pathway to Turning Genes into Medicine

Gene Therapy

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

Gene Therapy

Explore a timeline of important gene therapy milestones related to therapies for rare genetic diseases.

Contact Spark® Therapeutics

Request Medical Information
Get information about Spark products and therapeutic areas or request a discussion with a medical science liaison.
Call 1-866-647-7275

Speak with a representative, Monday through Friday from 8 AM to 8 PM ET.