Turning
complexity
into clarity

An understandable resource on gene therapy research

Gene Therapy Research

What are you curious about?

What Is gene therapy?

What are the steps involved with gene transfer therapy?

What causes genetic conditions?

What are the goals of gene transfer therapy research?

What are the possible risks of gene therapy research?

Upcoming Virtual Event

Drug Discovery and Beyond with Hemophilia Foundation of Michigan

Presented by:

Jonathan Bernstein, MD
Associate Professor, Director, Pediatric Hematology
Penn State University

08/17/22

View all upcoming events

Learn about a study BioMarin is conducting

The study aims to research rates of pre-existing immunity to certain naturally occurring viruses used in gene therapy research and how that changes over time.

Learn More

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