Question

In 1990 first test of human gene therapy was performed in 4-year-old girl ‘De Silva’ with adenosine deaminase deficiency.

1. Name the method used to cure the disease

2. Explain the procedure of r DNA technology used to recover adenosine deaminase deficiency

3. Which is the best cure for this disease?

Answer 1

1. Gene therapy. 

2. Lymphocytes are taken from patient’s blood it is grown invitro culture. By using retroviral vector functional ADA cDNA (from WBC of normal person) is introduced into vitro cultured lymphocytes These genetically engineered lymphocytes (corrected functional lymphocytes) are reinjected into the patients.

3. If the ADA producing genes are introduced into cells at early embryonic stage it could be a permanent cure.