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Adenosine Deaminase Deficiency

ADA- Immunodeficient Patients Successfully Complete Gene Therapy Protocols

Published in Gene Therapy Weekly, November 6th, 1995

Clinical trials of gene therapy protocols aimed at treating adenosine deaminase (ADA) deficiency show clinical success with long-term gene expression.

Severe combined immunodeficiency (SCID) as a result of inherited deficiency of ADA is usually fatal unless the affected children are kept in protective isolation or if their immune system is not reconstituted by bone marrow transplantation. Many patients do not have an HLA-identical sibling donor to make this particular cure possible. Alternative, hopefully curative therapies are desperately needed.

Italian researchers Claudio Bordignon et al. developed a clinical trial protocol to test a gene...

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