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| T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS |
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| Author(s): BLAESE RM, CULVER KW, MILLER AD, CARTER CS, FLEISHER T, CLERICI M, SHEARER G, CHANG L, CHIANG YW, TOLSTOSHEV P, GREENBLATT JJ, ROSENBERG SA, KLEIN H, BERGER M, MULLEN CA, RAMSEY WJ, MUUL L, MORGAN RA, ANDERSON WF |
| Source: SCIENCE Volume: 270 Issue: 5235 Pages: 475-480 Published: OCT 20 1995 |
| Times Cited: 557 References: 75 |
| Abstract: In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA(-) SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease. |
| Document Type: Article |
| Language: English |
| Reprint Address: BLAESE, RM (reprint author), NIH, NATL CTR HUMAN GENOME RES, BLDG 49, ROOM 2A03, BETHESDA, MD 20892 USA |
Addresses:
1. NCI, BETHESDA, MD 20892 USA
2. FRED HUTCHINSON CANC RES CTR, SEATTLE, WA 98104 USA
3. NIH, CTR CLIN, BETHESDA, MD 20892 USA
4. NHLBI, BETHESDA, MD 20892 USA
5. GENET THERAPY, GAITHERSBURG, MD 20878 USA
6. CASE WESTERN RESERVE UNIV, SCH MED, DEPT PEDIAT, CLEVELAND, OH 44106 USA |
| Publisher: AMER ASSOC ADVAN SCIENCE, 1333 H ST NW, WASHINGTON, DC 20005 |
| Subject Category: Multidisciplinary Sciences |
| IDS Number: TA374 |
| ISSN: 0036-8075 |
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| | | | | | | | | Record from Web of Science® | | | | | | | | | |