Gene therapy for ada deficiency

Enzyme-replacement therapy is effective in most patients with ADA deficiency but often fails to sustain lymphocyte counts and T-cell function. We found that ADA gene therapy improves immune function in patients who had insufficient immune reconstitution during PEG-ADA therapy. By using a unique type of gene therapy vector and treatment process, St. ... This genetic mutation affects white blood cells, mainly T cells, B cells and NK cells. These cells help the body fight off infections and protect against bacteria and other invaders. Adenosine deaminase deficiency is caused by mutations in the ADA gene. This gene provides instructions for producing the enzyme adenosine deaminase. This enzyme is found throughout the body but is most active in specialized white blood cells called lymphocytes.