Gene Therapy Consultation
- Monogenic Disorders
- Polygenic Disorders
- Infectious Diseases
Gene Therapy Makes Different Future
There are several diseases which is caused by one or more genetic alteration with no special treatment. These disorders could be congenital like Duchene syndrome and Thalassemia or acquired such as different kind of cancers. Because there is no specific cure for genetic diseases, the treatment strategy for these patients is maintenance therapy which is very costly and impose a huge cost on the health system followed by a lot of social impacts. Having this regard, many successful studies performed in order to investigate the possibility of whether genetic defect could be repaired via gene therapy.
Gene therapy can be defined as making changes to the genetic materials inside the nucleus of the cells which is called DNA in order to repair a defect responsible for a disorder. This modification performed using insertion of genetic materials into cells commonly via vectors. Certain modified viruses that can’t cause disease are often used as vectors because they can deliver the new gene by infecting the cell like retroviruses, adenovirus and adeno associated virus.
The most known gene therapy technology is Chimeric Antigen Receptor-T cells or CAR-T Cell therapy in which the white blood cells of the patient infected with the viruses ex-vivo. The viruses insert new gene to the T lymphocytes that the program then to produce the specific antigen receptors. These modified T-cells then multiplied in the laboratory and returned to the patient. Chemotherapy is used before reinjection of the engineered T-cells in order to prevention of rejection by the immune system. These new CAR T-Cells identify and destroy the specific cancerous cells which express this kind of receptors. Kymriah is the first CAR T-Cell therapy approved by FDA on August 2017 for the treatment of a special kind of ALL in patients under 25 years old. This was a promising step toward cure of the cancer, end-stage disease and rare diseases.