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| SUCCESSFUL EX-VIVO GENE-THERAPY DIRECTED TO LIVER IN A PATIENT WITH FAMILIAL HYPERCHOLESTEROLEMIA |
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| Author(s): GROSSMAN M, RAPER SE, KOZARSKY K, STEIN EA, ENGELHARDT JF, MULLER D, LUPIEN PJ, WILSON JM |
| Source: NATURE GENETICS Volume: 6 Issue: 4 Pages: 335-341 Published: APR 1994 |
| Times Cited: 389 References: 17 |
| Abstract: An ex vivo approach to gene therapy for familiar hypercholesterolaemia (FH) has been developed in which the recipient is transplanted with autologous hepatocytes that are genetically corrected with recombinant retroviruses carrying the LDL receptor. We describe the treatment of a 29 year old woman with homozygous FH by ex vivo gene therapy directed to liver. She tolerated the procedures well and in situ hybridization of liver tissue four months after therapy revealed evidence for engraftment of transgene expressing cells. The patient's LDL/HDL ratio declined from 10-13 before gene therapy to 5-8 following gene therapy, improvements which have remained stable for the duration of the treatment (18 months). This represents the first report of human gene therapy in which stable correction of a therapeutic endpoint has been achieved. |
| Document Type: Article |
| Language: English |
Addresses:
1. UNIV MICHIGAN, MED CTR, DEPT INTERNAL MED, ANN ARBOR, MI 48109 USA
2. UNIV MICHIGAN, MED CTR, DEPT SURG, ANN ARBOR, MI 48109 USA
3. UNIV MICHIGAN, MED CTR, DEPT BIOL CHEM, ANN ARBOR, MI 48109 USA
4. CHRIST HOSP, CARDIOVASC RES CTR, DIV LIPID METAB & PREVENT ATHEROSCLEROSIS, CINCINNATI, OH 45219 USA
5. CHRIST HOSP, CARDIOVASC RES CTR, MED RES LABS, CINCINNATI, OH 45219 USA
6. LAVAL UNIV HOSP, MED RES CTR, LIPID RES CTR, QUEBEC CITY G1V 4G2, PQ CANADA |
| Publisher: NATURE PUBLISHING CO, 345 PARK AVE SOUTH, NEW YORK, NY 10010-1707 |
| Subject Category: Genetics & Heredity |
| IDS Number: ND870 |
| ISSN: 1061-4036 |
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| | | | | | | | | Record from Web of Science® | | | | | | | | | |