Disease treated by Gene therapy Example A. Severe Combined Immunodeficiency (SCID) 1. Abnormal hemoglobin production B. Cystic Fibrosis 2. Defective CFTR gene C. Hemophilia 3. Deficiency of clotting factors D. Sickle Cell Anemia 4. Deficien

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Disease treated by Gene therapy Example A. Severe Combined Immunodeficiency (SCID) 1. Abnormal hemoglobin production B. Cystic Fibrosis 2. Defective CFTR gene C. Hemophilia 3. Deficiency of clotting factors D. Sickle Cell Anemia 4. Deficiency of adenosine deaminase

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Correct answer: B. A - 4, B - 2, C - 3, D - 1

SCID is caused by a deficiency of adenosine deaminase Cystic Fibrosis is due to a defective CFTR gene Hemophilia is caused by a deficiency of clotting factors. Sickle Cell Anemia is caused by abnormal hemoglobin production

Disease treated by Gene therapy Example A. Severe Combined Immunodeficiency (SCID) 1. Abnormal hemoglobin production B. Cystic Fibrosis 2. Defective CFTR gene C. Hemophilia 3. Deficiency of clotting factors D. Sickle Cell Anemia 4. Deficiency of adenosine deaminase

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