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| ENGRAFTMENT OF GENE-MODIFIED UMBILICAL-CORD BLOOD-CELLS IN NEONATES WITH ADENOSINE-DEAMINASE DEFICIENCY |
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| Author(s): KOHN DB, WEINBERG KI, NOLTA JA, HEISS LN, LENARSKY C, CROOKS GM, HANLEY ME, ANNETT G, BROOKS JS, ELKHOUREIY A, LAWRENCE K, WELLS S, MOEN RC, BASTIAN J, WILLIAMSHERMAN DE, ELDER M, WARA D, BOWEN T, HERSHFIELD MS, MULLEN CA, BLAESE RM, PARKMAN R |
| Source: NATURE MEDICINE Volume: 1 Issue: 10 Pages: 1017-1023 Published: OCT 1995 |
| Times Cited: 381 References: 31 |
| Abstract: Haematopoietic stem cells in umbilical cord blood are an attractive target for gene therapy of inborn errors of metabolism. Three neonates with severe combined immunodeficiency were treated by retroviral-mediated transduction of the CD34+ cells from their umbilical cord blood with a normal human adenosine deaminase complementary DNA followed by autologous transplantation. The continued presence and expression of the introduced gene in leukocytes from bone marrow and peripheral blood for 18 months demonstrates that umbilical cord blood cells may be genetically modified with retroviral vectors and engrafted in neonates for gene therapy. |
| Document Type: Article |
| Language: English |
| Reprint Address: KOHN, DB (reprint author), UNIV SO CALIF, CHILDRENS HOSP, SCH MED, DIV RES IMMUNOL BONE MARROW TRANSPLANTAT, 4650 SUNSET BLVD, LOS ANGELES, CA 90027 USA |
Addresses:
1. GENET THERAPY INC, GAITHERSBURG, MD 20878 USA
2. CHILDRENS HOSP & HLTH CTR, SAN DIEGO, CA 92123 USA
3. UNIV CALIF SAN FRANCISCO, DEPT PEDIAT, SAN FRANCISCO, CA 94143 USA
4. DUKE UNIV, MED CTR, DEPT MED, DURHAM, NC 27710 USA
5. NIH, NATL CTR HUMAN GENOME RES, BETHESDA, MD 20892 USA |
| Publisher: NATURE PUBLISHING CO, 345 PARK AVE SOUTH, NEW YORK, NY 10010-1707 |
| Subject Category: Biochemistry & Molecular Biology; Cell Biology; Medicine, Research & Experimental |
| IDS Number: RX951 |
| ISSN: 1078-8956 |
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