Open Navigation
Close Navigation
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 use of genetic material in the treatment or prevention of disease.
Vectors are essentially vehicles designed to deliver therapeutic genetic material, such as a working gene, directly into a cell.
There are many possible ways to target a disease or genetic variation, so there are different gene and cell therapy approaches that can be considered.
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.