MCQ
The first clinical gene therapy was given for treating
  • A
    diabetes mellitus
  • B
    chicken pox
  • C
    rheumatoid arthritis
  • adenosine deaminase deficiency.

Answer

Correct option: D.
adenosine deaminase deficiency.
d
(d) : 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 non­functional 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.

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