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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. Learn More 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. Learn More 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. Learn More