There are more than 4000 genetic disorders without an effective treatment modality. Majority of the treatments are symptomatic not considering the root cause of the disease. Many are either due to a faulty protein/ enzyme or by a defective gene.
Is it possible to provide correct version of a defective gene into a mammalian cells?
The development of gene transfer methods and rDNA technology allowed us to introduce genes into mammalian cells. This technological advancement constitutes the major step towards gene therapy.
What is gene therapy?
Gene therapy is a method that aims to cure inherited diseases by providing the patient with correct copy of the defective gene.
|Gene Therapy : How it works?|
Definition Gene therapy
The treatment of genetic diseases by introducing proper genes into patients's cells is called gene therapy.
There are four potential approaches to gene therapy:
1) Addition of normal gene to replace the function of defective gene. This is gene replacement or gene augmentation therapy.
2) Replacing the defective gene with the correct gene. This is Corrective gene therapy.
3) Establishment of alternative pathways that bypass mutant genes function
4) Change in regulation of normal or mutant genesThe first two are the basic approaches in gene therapy