Question

What is gene therapy? Explain the steps involved in curing ada deficiency by gene therapy. 2?

Answer 1

We conducted a gene therapy trial in 10 patients with adenosine deaminase (ADA)–deficient severe combined immunodeficiency using 2 slightly different retroviral vectors for the transduction of patients' bone marrow CD34+ cells.

Answer 2

Gene therapy and Steps involved in curing ADA deficiency by gene therapy:

• Gene therapy is the tool in medical treatment which involves the introduction of genetic material into the target cells to cure the particular disease.

• Adenosine deaminase (ADA) deficiency is genetically transferred (inherited) disorder which destroys the immune system and results in severe immunodeficiency.

• The symptoms of ADA deficiency  are pneumonia, chronic diarrhea and skin rashes.

• Retrovirus mediated gene transfer is used to treat adenosine deaminase (ADA) deficiency.

Steps involved:

• Incorporation of ADA gene into genetically disabled retrovirus.

• Isolation of T-cell stem line cells with disabled ADA gene from patient  and exposed to cell culture with retrovirus.

• During infection, retrovirus transfers ADA gene to cells.

• Identification of transformed cells.

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