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Adenosine deaminase deficiency or ADA

Adenosine deaminase deficiency  is a severe immunodeficiency disease that results from a lack of the enzyme adenosine deaminase. It usually leads to death within the first few months of life. Adenosine deaminase is an enzyme that plays a key role in salvaging purine molecules.

Treatment of Adenosine deaminase deficiency, or ADA deficiency

  • Bone marrow or stem cell transplants from a haploidentical (half-matched) donor is available for a minority of patients.

  • Enzyme therapy can directly add missing adenosine deaminase enzyme.

  • Somatic Gene therapy is provided by introducing a new therapeutic gene (transgene) into the diseased cells of a patient. The modified cells express the introduced gene and their new phenotype provides some advantage to the patient. For this approach two prerequisites must be worked out:

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