Gene therapy for ADA deficiencyADA की कमी के लिए जीन चिकित्सा

Class 12 Biology · Chapter 10 · interactive diagram

Parts and their functions

• Patient with ADA deficiency (ADA की कमी वाला रोगी) — First clinical gene therapy (1990): a 4-year-old girl with adenosine deaminase deficiency. The disorder is caused by DELETION of the ADA gene; the enzyme is crucial for the immune system.
• Lymphocytes from blood (रक्त से लसीकाणु) — Lymphocytes are taken from the patient’s blood.
• Culture outside the body (शरीर के बाहर संवर्धन) — The lymphocytes are grown in a culture outside the body.
• ADA cDNA + retroviral vector (ADA cDNA + रेट्रोवायरस वाहक) — A functional ADA cDNA is introduced into the lymphocytes using a retroviral vector.
• Cells returned to the patient (कोशिकाएँ रोगी में वापस) — The genetically engineered lymphocytes are put back into the patient.
• Periodic infusion (समय-समय पर निवेशन) — These cells are not immortal, so the patient needs periodic infusions of engineered lymphocytes.
• Permanent cure (स्थायी उपचार) — If the ADA gene isolated from marrow cells is introduced into cells at early embryonic stages, it could be a permanent cure.

Steps

1. Lymphocytes are taken from the patient’s blood.
2. They are grown in culture outside the body.
3. A functional ADA cDNA is introduced using a retroviral vector.
4. The engineered lymphocytes are returned to the patient.
5. The cells are not immortal — infusion must be repeated periodically.
6. A permanent cure: ADA gene (from marrow cells) into early embryonic cells.

Original diagram drawn by Simple Learning Hub, based on the NCERT textbook. Report a mistake