What are gene therapy's goals?

Gene therapy is a rapidly evolving form of molecular medicine with the potential to provide new treatments for inherited and acquired life-threatening rare diseases. When a gene is damaged, the blueprint for the construction of the protein it codes for is flawed, resulting in an abnormal protein and a genetic disease. read more

The ideal situation would be to treat or even cure inherited diseases and disorders. The ideal candidates are those which are caused by a single gene, even though most disorders are the result of multiple gene interactions. read more

To achieve this goal, gene therapy requires the technology to insert the missing “normal” gene into the DNA of the patient’s cells. One promising approach relies on the use of modified viruses called “viral vectors” to carry the “normal” gene into cell and into the subject’s DNA. read more