Gene Therapy

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. The goal of gene therapy is to cure or slowdown a genetic disease by repairing the damaged gene responsible for the disease. 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. The “normal” gene then replicates and is passed on when the cell divides. The fundamental concept of viral vectors comes from the innate ability of viruses to deliver genetic material into an infected cell. Researchers have discovered ways to manipulate the genome of the virus to remove the disease causing genetic components and incorporate the replacement human gene.

Worldwide over 600 clinical trials using gene therapy have been conducted or are currently underway. Gene transfer into multi-potent hematopoietic bone marrow cells has received much attention because of the broad variety of human diseases and the relative ease to achieve gene transfer into the diseases cells. Gene transfer by these viral vectors has been under clinical study for more than 15 years with resulting success.

The use of gene therapy has been recognized as a potential curative treatment for the blood disorder thalassemia. This therapy is being developed under the trade name Thalagen™.