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Gene Therapy- An emerging tool of Biotechnology

Gene Therapy is the insertion of gene and biological tissues in the human body to treat disease such as cancer. This technique is very new and is still in its infancy, not ready to shower its blessings on us.But scientific breakthrough will take it a miles ahead and will make it a grand success towards the mainstream medicine.

The first approved gene therapy took place in United States at Sept14, 1990 in the National Institute of Health.It was performed on a four year old girl for genetic defect that led to immune system deficiency.

Sickle cell disease was successfully treated on mice through this disease.Moreover,blood disorder like thalassemia was also treated through this technique.

In most gene therapy studies, normal gene is inserted in order to replace the abnormal gene.A carrier molecule must be present to deliver the gene into patient’s target cells.Currently the carrier is a virus which is genetically altered to carry normal human DNA. Target cells such as patients liver or lung cells are infected with the viral vector.The vector then unloads its genetic material containing the therapeutic human gene into its target cell.Some of the different gene types of viruses used as gene therapy vectors are- retrovirus,adenovirus, herpes simplex virus.

Besides virus mediated gene therapy systems there are non viral options available too.The simplest method is the direct introduction of therapeutic DNA into its target cells.The approach is limited in its application as it can only be used where there is huge amount of DNA.

Some recent developments in Gene therapy research are to combine Nanotechnology along with gene therapy to target an destroy hard-to-reach cancer cells.Moreover, world’s finest gene therapy in Uk for inherited blindness has shown successful results in sight improvement.

Gene Thearpy will prove its far reaching effects to cure incurable diseases like Huntington Chorea within a few more years.Gene therapy will make RNA silencing a more advanced tool and thus will help the mankind.