Gene therapy involves technique of replacing absent or defective gene by a corrective gene, so that the body can make the correct enzyme or protein and consequently eradicate the root cause of genetic disease.
A gene can be defined as an area of DNA that controls genetic characteristics of a person. Genes contains the information to build and maintain their cells and pass genetic attributes to offspring. Genes also determine person’s sex, physical trait and IQ. Scientists believe that every individual has about 25,000 genes per cell. A genetic mutation, or alteration, in any one of these genes can result in hereditary disease, physical impairment, or shortened life span. This genetic mutation can be passed on from one generation to another. By gene therapy one can eliminate these changes by replacing faulty gene by genetically engineered gene.
Gene therapy also holds promise for people with HIV-AIDS, cancer, muscular dystrophies, hemophilia A, and certain neurological disorder like Parkinson's disease and Alzheimer’s disease. The most active research being done in gene therapy for children has been for genetic disorders like cystic fibrosis and adenosine deaminase deficiency ( a rare genetic disorder that makes kid vulnerable to serious infection), and those with hypercholesterolemia( extremely high level of serum cholesterol).
To cure genetic diseases, scientist must first find out which gene or set of genes causes such diseases. The Human Genome Project in the US and other international efforts have completed the initial work of sequencing and mapping of all of the 25,000 to 35,000 genes in human cell. It is hoped that in 15 years time scientists will be able to identify, diagnose, treat and cure all diseases to which humans are susceptible. This research will revolutionize modern medication and hopefully ameliorate the quality of life of all human beings suffering from hemophilia.
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