Gene Delivery for Serious Diseases

Gene therapy is a technique used to treat serious disease by adding a gene to a person’s cells. The added gene may be a healthy copy of a mutated gene. It may also be a different gene that inactivates or compensates for the disease-causing gene.

One example is severe combined immunodeficiency, or...

Gene therapy is the insertion of a gene into an individual's cells to prevent or treat serious diseases. The inserted gene may be a healthy copy of a mutated gene, or it may be a different gene that counteracts the effects of a faulty gene.

Genes can be delivered in vivo, directly into a person, or ex vivo, into cells removed from the bo...