Inventors:
Donald B. Kohn - Tarzana CA, US
R. Michael Blaese - Rockville MD, US
Craig A. Mullen - Sugar Land TX, US
Robert C. Moen - Mountain View CA, US
Assignee:
Children's Hospital of LosAngeles - LosAngeles CA
The United States of America as represented by the Department of Health and Human Services - Washington DC
International Classification:
A61K 48/00, C12N 15/63, C12N 15/74, C12N 15/00, C12N 5/00
US Classification:
424 9321, 424 932, 514 44, 435325, 4353201, 4352401, 4352402, 530350, 536 221, 536 231, 536 241
Abstract:
A method of providing a therapeutic effect in a human patient which comprises administering to the patient CD34+ cells obtained from cord blood. The CD34+ cells have been engineered with at least one nucleic acid sequence encoding a therapeutic agent. Such CD34+ cells may be engineered by transducing the cells with a retroviral vector including the nucleic acid sequence encoding the therapeutic agent. This method has been applied in treating newborn infants suffering from ADA deficiency.