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Question
State the steps involved in the process of gene therapy for the treatment of ADA-deficiency.
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Solution
- Stem cells from the bone marrow, responsible for producing lymphocytes in ADA-deficient patients, are extracted. These cells harbor the faulty ADA gene.
- A retrovirus, a specialized RNA virus, serves as a vector in this process.
- The normal ADA gene is replicated through recombinant DNA technology and incorporated into the nucleic acid of a retrovirus, engineered to be non-pathogenic.
- The bone marrow stem cells from ADA-deficient individuals undergo infection with the retrovirus.
- The virus introduces its nucleic acid (rDNA), which includes the functional ADA gene, into the chromosome of the stem cell.
- This provides these bone marrow stem cells with a functional copy of the gene alongside the pre-existing defective gene.
- The treated stem cells continue to replicate over the patient's lifetime. Possessing a fully functional ADA gene, they produce the ADA enzyme. These modified cells supply the lacking enzyme, leading to the cure of the ADA-deficient patient.
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