Treatment of adenosine deaminase (ADA) deficiency is an exampleof ex vivo gene therapy. Why is this therapy called ex vivo?Can ex vivo gene therapy be used to treat all inherited diseases?Explain.
Treatment of adenosine deaminase (ADA) deficiency is an example
of ex vivo gene therapy. Why is this therapy called ex vivo?
Can ex vivo gene therapy be used to treat all inherited diseases?
Explain.
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 hereditary disease. Correction of a
genetic defect involves compensation for the non-functional gene by a normal gene. First clinical gene therapy was
given in 1990 to a 4-year old girl with adenosine deaminase (ADA) deficiency. The disorder is caused due to the deletion
of the gene for adenosine deaminase, the enzyme crucial for the immune system to function.
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