ASGCT News 

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FDA Approves First Gene Therapy for AADC Deficiency

Paula Cannon, PhD

Here's What to Know Before #ASGCTBreakthroughs24

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Advancing knowledge, awareness, + education of gene and cell therapies

About ASGCT

2024

Breakthroughs in Muscular Dystrophy

November 19-20, 2024 | Chicago, IL

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Gene & Cell Therapy Clinical Trials

Browse our curated database of gene and cell therapy clinical trials from throughout the United States. Search by type of therapy, diagnosis, location of trial, and more.

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Educational Resources

Learn the basic science of gene therapy, cell therapy, and gene editing

Gene Therapy Basics

Gene therapy has been studied for more than 40 years and can help stop or slow the effects of disease on the most basic level of the human body—our genes. And to understand how it works, we’ll start at the basics.

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Gene therapy, cell therapy, and gene editing all have the same goal, but use different routes to achieve it

Gene Therapy Approaches

With many possible ways to target a disease or genetic variation, scientists consider different gene and cell therapy approaches. Learn more about some of these strategies from the lab or already approved for use in humans.

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How do clinical trials work? Follow gene therapy from the laboratory to a patient

Developing a Treatment

Gene therapies take years to go from theoretical concepts, to preclinical trials, to clinical trials, and, finally, into actual treatments that can improve the lives of patients. Here we learn about the process of developing a treatment.

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What is ASGCT?

The American Society of Gene & Cell Therapy (ASGCT) is the primary membership organization for scientists, physicians, professionals, and patient advocates involved in gene and cell therapy. Our mission is to advance knowledge, awareness, and education leading to discovery and clinical application of gene and cell therapies to alleviate disease—we do that by serving as a catalyst to transform medicine by advancing gene and cell therapies to benefit patients and society.

Become a Member

ASGCT Membership
ASGCT membership is essential to scientists working in gene and cell therapy

Membership Benefits:

  • Education & Training
  • Advocacy Opportunities
  • Career Growth
  • Reduced Annual Meeting Fees
  • Free Molecular Therapy Subscription

Become a Member

Molecular Therapy:

The International Journal of Molecular and Cellular Therapeutics

About the Journal

Official Journal of the American Society of Gene & Cell Therapy

Molecular Therapy is the leading journal for gene and cell therapy research, publishing important peer-reviewed research and cutting-edge reviews and commentaries.

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