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Gene Therapy Field Advances With Important Clinical Findings Reported in the New England Journal of Medicine
Date:2/3/2009

SAN DIEGO, Calif., Feb. 3 /PRNewswire-FirstCall/ -- Cardium Therapeutics (NYSE: CXM) today reported on recent clinical findings published in The New England Journal of Medicine showing the long-term outcome of a method of gene therapy to treat infant children with a fatal disorder of the immune system. The infant children studied had a rare form of severe combined immunodeficiency (SCID). SCID arises in infants with a genetic defect that leaves them unable to produce an enzyme called adenosine deaminase. This key enzyme is most active in lymphocytes, which are white blood cells vitally important for protection against infection by bacteria, viruses and fungi. Infants who inherit this fatal genetic abnormality develop impaired immune responses, leading to recurrent infections and a failure to thrive. Without adequate treatment, these children typically die within one year of birth.

(Logo: http://www.newscom.com/cgi-bin/prnh/20051018/CARDIUMLOGO)

The New England Journal of Medicine (NEJM) reported on the success of a DNA-based approach for the introduction of a normal adenosine deaminase gene into the patient's hematopoietic stem cells that have been removed from the bone marrow. After transfection of the cells with the gene, the cells are re-infused back into the patients after nonmyeloablative conditioning with busulfan. After a single treatment, all 10 children in the study survived and gene therapy was not associated with any adverse events during a median follow-up of 4 years. In 8 of the 10 patients, there was excellent and persistent immune reconstitution and protection from infection.

In an accompanying NEJM editorial entitled, Gene Therapy Fulfilling Its Promise, the authors noted, "The prospects of cont
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SOURCE Cardium Therapeutics
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