Gene Therapy

Gene therapy is a medical treatment that works by adding, removing, or modifying genes in a person’s cells to treat or prevent diseases.

There are two main types of gene therapy.

In vivo procedures are performed inside the body, while ex vivo procedures are carried out outside the body.

In vivo therapy delivers corrected genes into the patient’s body using vectors— carriers that deliver genes to a specific target.

For example, to treat cystic fibrosis, a genetic disorder, a solution containing the vector with a healthy copy of the gene can be sprayed into the patient’s nose.

This helps the lungs produce thinner mucus, improving breathing and reducing infections.

In ex vivo therapy, doctors remove cells from the patient, repair the faulty gene, and return the healthy cells to the body.

This method treats diseases like sickle cell anemia, where bone marrow cells are taken from the patient, the faulty genes are corrected, and the cells are placed back into the patient.