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- What is Gene therapy – Illustrate using example of Adenosine deaminase deficiency?Which kind of therapy was given in 1990 to a four year old girl with adenosine deaminase (ADA) deficiency?The first person to receive gene therapy was a young girl with adenosine desminase(ADA) deficiency. Outline the therapeutic steps involved in this gene therapy.
- What is c3435T mutataion,What is the clinical implication of c3435T mutation, what is the molecular characteristics of c3435T mutaionWhat percentage of cells in an organ or a tissue need toexpress a therapeutic gene to alleviate the effects of agenetic disorder?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.