Q.
The first clinical gene therapy was given for treating
3169
206
AIPMTAIPMT 2012Biotechnology and its Applications
Report Error
Solution:
Gene therapy is a collection of methods that allows correction of a gene defect that has been diagnosed in a child/embryo. Here genes are inserted into a person's cells and tissues to treat a disease. Correction of a genetic defect involves delivery of a normal gene into the individual or embryo to take over the function of and compensate for the non-functional gene. The first clinical gene therapy was given in 1990 to a 4 - year old girl with adenosine deaminase (ADA) deficiency. This enzyme is very important for the immune system to function. SCID is caused due to defect in the gene for the enzyme adenosine deaminase. In some children ADA deficiency can be cured by bone marrow transplantation. Here, the isolated gene from bone marrow cells producing ADA is introduced into cells at early embryonic stages; it can be a permanent cure.