Gene therapy is a medical technique that attempts to manipulate or modify a gene’s expression or to alter the biological properties of living cells. Changing these genetic components of an individual serves as a medical approach to treat or prevent diseases and may be used in place of surgery or medication.
One gene therapy method is gene addition, which introduces a new gene into cells that allows the body to fight diseases. Gene transfer is another method in gene therapy that presents a non-faulty copy of a gene to replace an altered and disease-causing gene.
A newer technique within gene therapy is genome editing, which introduces molecular tools to change existing cell DNA. This may help turn genes on/off or fix genetic alterations that underlie a disorder.
Various gene therapy products include:
- viral vectors
- plasmid DNA
- human gene editing technology
- bacterial vectors
- patient-derived cellular gene therapy products