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.
