Q12 of 50 Page 1

What is gene therapy? Name the first clinical case where it was used.

Gene therapy is a accumulation of methods which allows for the correction of a genetic defect that has been diagnosed in a child/embryo.

In gene therapy, normal genes are inserted into a person’s cells and tissues to treat a particular genetic disease or for treating non-functional genes.


The first clinical case where it was used was in the year 1990 where a 4-year old girl with adenosine deaminase (ADA) deficiency was diagnosed. The enzyme, adenosine deaminase is crucial for the immune system to function properly. Few of the procedures to cure this is by bone marrow transplantation or by enzyme replacement therapy.


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