Gene & Cell Therapy Education | ASGCT - American Society of Gene & Cell Therapy |

Gene & Cell Therapy 101

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.

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Disease Treatments

Learn more about how gene therapy works for specific diseases.

Hemophilia

Hemophilia occurs when the genes that are vital to producing clotting factors are faulty.

Fabry Disease

Gene therapy approaches target a faulty GLA gene.

Batten Disease

Different forms of the disease being researched for potential treatments include CLN1, 2, 3, 5, 6, 7, and 8.

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ASGCT Clinical Trials Finder

Use this interactive database to find all active and recruiting gene and cell therapy clinical trials around the world.

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Patient Journey

Helpful information to guide you through the challenging journey from diagnosis to pursuing a gene therapy.

Identifying a Genetic Disease

Genetic testing, in a variety of forms, is one way to identify rare genetic diseases.

Next Steps With a Diagnosis

Here are information and resources to take some first steps after you've received a confirmed diagnosis of a rare genetic disorder.

Stimulating Research

Patients and advocates play an important role to initiate gene therapy research.

Considering a Clinical Trial

Here's a glimpse into using the clinical trials finder and what to expect when entering a clinical trial.

Ethical Issues: Germline Gene Editing

This page addresses key ethical questions that patients, their families, and the public might have about this technology.

Ethical Issues: Unproven Stem Cell Products

Learn about some of the signs that might help you decide whether a provider of cell therapies can be trusted.

Patient Education Program Wins National Award

We are thrilled to announce that ASGCT has been named a winner of the 2021 "Power of A" Silver Award from the American Society of Association Executives (ASAE) for our Patient Education Program!

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