Gene therapy in the treatment of diseases

Gene therapy' involves inserting one or more corrected genes into apatient's genetic material to treat a genetic disease. The genes are designedin the lab to compensate for errors in the patient's DNA. Therefore, theexpression of the new gene or genes can alter the DNA or RNA transcriptused to synthesize proteins, thereby correcting the disease. In spite of itspotential, gene therapy is still in its infancy and not widely used yet. Genetherapy using an adenovirus vector: A new gene is injected into anadenovirus vector, which is used to introduce the modified DNA into ahuman cell. If the treatment is successful, the new gene will make afunctional protein. Procedure of gene therapy: Some of the main diseasescurrently being researched in gene therapy studies include: Cystic fibrosis,Hemophilia, Muscular dystrophy, Cancer. One of the main vectors used tocarry modified genes into cells is the virus. Viruses usually attach to a hostcell and transfer their viral genetic material into it. They then take over thecell and use the cell components to make copies of the virus. Scientists canmodify these viruses so that they contain only therapeutic and not viralgenetic material but still retain their infective ability so that the beneficialDNA can be transferred into host cells. Commonly used vectors includeadenoviruses, adeno-associated viruses, retroviruses and the herpes simplexvirus. In this article, the application of gene therapy in the treatment ofdiseases has been investigated.

Keywords:

Gene therapy , Genetic disease , Cystic fibrosis , Hemophilia , Cancer