Gene Therapy Introduction

Introduction

- Mr.V.G.Prem Kumar

• Gene therapy is a therapeutic procedure which is used to treat Inherited and diseases caused by mutation in DNA and disease caused by infectious diseases. Gene therapy is a very valuable procedure used in cases where there is no conventional treatment available.
• In Gene therapy, Gene transfer is done, in which therapeutic genes are transferred into host cells, in order to modify the genetic information in the cells of the patient.
• Genes have multiple functions to play for proper functioning of an organism, so mutation in a single gene may cause many biological consequences.
• Gene mutation may cause an alteration in enzyme activity causing either an accumulation or deficiency of a substrate, leading to malfunctioning of the normal metabolism. Mutation in a gene coding protein structure may cause cell tissue or organ abnormalities.
• Genetic mutations can cause disorders in every part of the human body including muscles, eyes, liver, bones, kidneys, heart, nerves, skin, brain, stomach, intestines, and blood systems.
• Defect in a gene expressed in one tissue may also cause abnormalities in another organ. The primary goal of human medical genetics research is to develop treatments for different genetic diseases.