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Scientific, reliable information for patients and the public
Gene therapy has been studied for more than 40 years and can stop or slow the effects of disease on the most basic level of the human body—our genes.
Learn more about how gene therapy works for specific diseases.
Hemophilia occurs when the genes that are vital to producing clotting factors are faulty.
Gene therapy approaches target a faulty GLA gene.
Different forms of the disease being researched for potential treatments include CLN1, 2, 3, 5, 6, 7, and 8.
Use this interactive database to find all active and recruiting gene and cell therapy clinical trials around the world.
Helpful information to guide you through the challenging journey from diagnosis to pursuing a gene therapy.
Genetic testing, in a variety of forms, is one way to identify rare genetic diseases.
Here are information and resources to take some first steps after you've received a confirmed diagnosis of a rare genetic disorder.
Patients and advocates play an important role to initiate gene therapy research.
Here's a glimpse into using the clinical trials finder and what to expect when entering a clinical trial.
This page addresses key ethical questions that patients, their families, and the public might have about this technology.
Coming soon!
The American Society of Gene & Cell Therapy is the primary professional membership organization for gene and cell therapy.