Question

Which pair consists of alternative treatments for ADA deficiency?

Options

• Haemophilia and cystic fibrosis

• Gene Augmentation Therapy and Gene Inhibition Therapy

• Bone marrow transplantation and enzyme replacement therapy

• Ex vivo delivery and in vivo delivery

Answer 1

Bone marrow transplantation and enzyme replacement therapy are alternative treatments for ADA deficiency. They are distinct from the procedure using genetically corrected lymphocytes.