Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID)
B Chan, D Wara, J Bastian, MS Hershfield… - Clinical …, 2005 - Elsevier
Adenosine deaminase (ADA)-deficient Severe Combined Immunodeficiency (ADA-deficient
SCID) is characterized by impaired lymphocyte development and function resulting from the
adenosine metabolism defect. Enzyme replacement therapy with polyethylene glycol-
conjugated adenosine deaminase (PEG-ADA) minimizes infectious complications of ADA-
deficient patients who have not received bone marrow transplantation. In PEG-ADA therapy,
enzymatically active ADA continuously circulates to act as a metabolic sink, detoxifying the …
SCID) is characterized by impaired lymphocyte development and function resulting from the
adenosine metabolism defect. Enzyme replacement therapy with polyethylene glycol-
conjugated adenosine deaminase (PEG-ADA) minimizes infectious complications of ADA-
deficient patients who have not received bone marrow transplantation. In PEG-ADA therapy,
enzymatically active ADA continuously circulates to act as a metabolic sink, detoxifying the …