Gene Therapy

If a person is born with a genetic disease, is there a cure for the disease?  

Gene therapy is one such attempt.  Gene therapy involves methods that correct genetic defects diagnosed in a baby or fetus.  

In which genes are inserted into a person's cells or tissues to treat the disease.  

The treatment of a genetic defect involves the insertion of common genes into an individual or a fetus.  

Which completes its functions by compensating for inactive genes.  

Gene therapy was first used in 1990 to treat adenosine deaminase (ADA) deficiency in a four-year-old girl.  

These enzymes are essential in the immune system.  This problem is caused by the deletion of the gene responsible for adenosine deaminase.  

In some children ADA is treated with bone marrow transplantation, while in others it is treated with enzyme replacement therapy in which active ADA is given to the patient through a syringe.  

The limitation of both of the above actions is that they are not completely antidote.  

Gene therapy first involves the extraction of lymphocytes from a patient's bloodstream.  

Active ADA - cDNA (using retrovirus carriers) is inserted into the lymphocytes which is then re-inserted into the patient's body. 

However, these cells are not immortal, so genetically engineered lymphocytes need to be inserted into the patient's body periodically. 

However, if the gene producing ADA isolated from the brain cells is introduced into the cells in the early embryonic stage, permanent cure is possible.