Safety and feasibility of liver-directed ex vivo gene therapy for homozygous familial hypercholesterolemia

Annals of Surgery

Volumn 223, Issue 2, 1996, Pages 116-126

  Raper, Steven E ^a   Grossman, Mariann ^a   Rader, Daniel J ^a   Thoene, Jess G ^b   Clark III, Bernard J ^c   Kolansky, Daniel M ^a   Muller, David W M ^b   Wilson, James M ^a  

^a WISTAR INSTITUTE   (United States)

^b UNIVERSITY OF MICHIGAN HEALTH SYSTEM   (United States)

^c HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; LOW DENSITY LIPOPROTEIN RECEPTOR;

ADULT; ARTICLE; AUTOSOMAL DOMINANT DISORDER; CHILD; CLINICAL ARTICLE; CLINICAL FEATURE; CLINICAL TRIAL; FAMILIAL HYPERCHOLESTEROLEMIA; FEMALE; GENE MUTATION; GENE THERAPY; GENETIC TRANSDUCTION; HOMOZYGOSITY; HUMAN; HUMAN CELL; LIVER CELL CULTURE; LIVER RESECTION; MALE; PORTAL VEIN; PRIORITY JOURNAL; RECEPTOR GENE; VEIN CATHETERIZATION; VIRUS RECOMBINANT;

ADULT; CANADA; CHILD; CHINA; COLOMBIA; COMBINED MODALITY THERAPY; CYPRUS; FEASIBILITY STUDIES; FEMALE; GENE THERAPY; HEPATECTOMY; HOMOZYGOTE; HUMANS; HYPERLIPOPROTEINEMIA TYPE II; LIVER; MALE;

EID: 0030022230     PISSN: 00034932     EISSN: None     Source Type: Journal    
DOI: 10.1097/00000658-199602000-00002     Document Type: Article

References (18)

1. Brown MS, Goldstein JL. A receptor-mediated pathway for cholesterol homeostasis. Science 1986; 232(4746):34-47.
2. Goldstein JL, Brown MS. Familial hypercholesterolemia: lipoprotein and lipid metabolism disorders. In Scriver CR, Beaudet al, Sly WS, Valle D, eds. Metabolic Basis of Inherited Disease. 6th ed. Vol 1. New York: McGraw-Hill, 1989, pp 1215-1250.
3. Starzl TE, Bahnson HT, Hardesty RL, et al. Heart-liver transplantation in a patient with familial hypercholesterolemia. Lancet 1984:1382-1383.
4. Bilheimer DW, Goldstein JL, Grundy SM, Starzl TE. Liver transplantation to provide low density lipoprotein receptors and lower plasma cholesterol in a child with homozygous familial hypercholesterolemia. N Engl J Med 1984; 311:1658-1664.
5. Wilson JM, Grossman M, Raper SE, et al. Clinical protocol: ex vivo gene therapy of familial hypercholesterolemia. Hum Gene Ther 1992; 3:179-222.
6. Goldstein JL, Kita T, Brown MS. Effective lipoprotein receptors and atherosclerosis. N Engl J Med 1983; 309:288-296.
7. Wilson JM, Roy Chowdhury N, Grossman M, et al. Temporary amelioration of hyperlipidemia in low density lipoprotein receptor-deficient rabbits transplanted with genetically modified hepatocytes. Proc Natl Acad Sci (USA) 1990; 87:8437-8441.
8. Roy Chowdhury J, Grossman M, Gupta S, et al. Long-term improvement of hypercholesterolemia after ex vivo gene therapy in LDLR-deficient rabbits. Science 1991; 254:1802-1805.
9. Grossman M, Raper SE, Wilson JM. Towards liver-directed gene therapy: Retrovirus-mediated gene transfer into human hepatocytes. Som Cell Mol Genet 1991; 17:601-607.
10. Raper SE, Wilson JM, Grossman M. Retroviral-mediated gene transfer in human hepatocytes. Surgery 1992; 112:333-340.
11. Grossman M, Wilson JM, Raper SE. A novel approach for introducing hepatocytes into the portal circulation. J Lab Clin Med 1993; 121:472-478.
12. Grossman M, Raper SE, Wilson JM. Transplantation of genetically-modified autologous hepatocytes into non-human primates: feasibility and short-term toxicity. Hum Gene Ther 1992; 3:501-510.
13. Grossman M, Raper SE, Kozarsky K, et al. Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolemia. Nature Genet 1994; 6:335-341.
14. Grossman M, Rader DJ, Muller D, et al. A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolemia. Nature Med 1995; 1:1148-1154.
15. Memsic L, Busuttil RW, Traverse LW. Bleeding esophageal varices and portal vein thrombosis after pancreatic mixed-cell autotransplantation. Surgery 1984; 95:238-242.
16. Scharp DW, Lacy PE, Santiago JV, et al. Results of our first nine intraportal islet autografts in type 1 insulin-dependent diabetic patients. Transplantation 1991; 51:76-85.
17. Taylor I, Machin D, Muller M, et al. A randomized controlled trial of adjuvant portal vein cytotoxic perfusion in colorectal cancer. Br J Surg 1985; 72:359-363.
18. Fielding LP, Hittinger R, Grace RH, Fry JS. Randomized controlled trial of adjuvant chemotherapy by portal-vein perfusion after curative resection for colorectal adenocarcinoma. Lancet 1992; 340:502-506.