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Gene therapy can stop or slow the effects of disease on the most basic level of the human body—our genes.
Gene therapy is the introduction, removal or change in genetic material—specifically DNA or RNA—into the cells of a patient to treat a specific disease.
Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell.
Gene therapy, cell therapy, and gene editing are fields of biomedical research with a similar goal in mind: To treat disease by changing our bodies at a microscopic level.
How do clinical trials work? Here we learn about the process of developing a treatment.
ASGCT and the National Organization for Rare Disorders (NORD) have partnered to produce a series of five patient education webinars.
Visit these websites for more gene and cell therapy resources.