Human thyroxine binding globulin (TBG) promoter directs efficient and sustaining transgene expression in liver-specific pattern

Gene

Volumn 506, Issue 2, 2012, Pages 289-294

  Yan, Zhonghai ^a   Yan, Hao ^b   Ou, Hailong ^c  

^a Och Spine at New York Presbyterian Hospitals   (United States)

^b ZHEJIANG UNIVERSITY SCHOOL OF MEDICINE   (China)

^c GUIZHOU MEDICAL UNIVERSITY   (China)

Author keywords

1 Antitrypsin; Gene therapy; Lentivirus vector; Liver; Promoter; Thyroxine binding globulin

Indexed keywords

ALPHA 1 ANTITRYPSIN; APOLIPOPROTEIN E; CYTOCHROME P450; ELONGATION FACTOR 1ALPHA; PHOSPHOGLYCERATE KINASE; THYROXINE BINDING GLOBULIN;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; GENE DOSAGE; GENE EXPRESSION; GENE FUNCTION; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; PRIORITY JOURNAL; TRANSGENE;

ALPHA 1-ANTITRYPSIN; ANIMALS; GENE EXPRESSION PROFILING; GENE EXPRESSION REGULATION; GENE THERAPY; HEP G2 CELLS; HUMANS; LIVER; MICE; MICE, INBRED C57BL; PROMOTER REGIONS, GENETIC; THYROXINE-BINDING GLOBULIN; TIME FACTORS; TRANSCRIPTION, GENETIC; TRANSFECTION; TRANSGENES;

LENTIVIRUS;

EID: 84864533194     PISSN: 03781119     EISSN: 18790038     Source Type: Journal    
DOI: 10.1016/j.gene.2012.07.009     Document Type: Article

References (21)

1. Al-Dosari M., Zhang G., Knapp J.E., Liu D. Evaluation of viral and mammalian promoters for driving transgene expression in mouse liver. Biochem. Biophys. Res. Commun. 2006, 339:673-678.
2. Bish L.T., Sleeper M.M., Forbes S.C., Morine K.J., Reynolds C., Singletary G.E., Trafny D., Pham J., Bogan J., Kornegay J.N., Vandenborne K., Walter G.A., Sweeney H.L. Long-term systemic myostatin inhibition via liver-targeted gene transfer in golden retriever muscular dystrophy. Hum. Gene Ther. 2011, 22:1499-1509.
3. Carrillo-Carrasco N., Chandler R.J., Chandrasekaran S., Venditti C.P. Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction. Hum. Gene Ther. 2010, 21:1147-1154.
4. Cotugno G., Annunziata P., Tessitore A., O'Malley T., Capalbo A., Faella A., Bartolomeo R., O'Donnell P., Wang P., Russo F., Sleeper M.M., Knox V.W., Fernandez S., Levanduski L., Hopwood J., De Leonibus E., Haskins M., Auricchio A. Long-term amelioration of feline mucopolysaccharidosis VI after AAV-mediated liver gene transfer. Mol. Ther. 2011, 19:461-469.
5. Gao G.P., Alvira M.R., Wang L., Calcedo R., Johnston J., Wilson J.M. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc. Natl. Acad. Sci. U. S. A. 2002, 99:11854-11859.
6. Haas D.L., Case S.S., Crooks G.M., Kohn D.B. Critical factors influencing stable transduction of human CD34(+) cells with HIV-1-derived lentiviral vectors. Mol. Ther. 2000, 2:71-80.
7. Hayashi Y., Mori Y., Janssen O.E., Sunthornthepvarakul T., Weiss R.E., Takeda K., Weinberg M., Seo H., Bell G.I., Refetoff S. Human thyroxine-binding globulin gene: complete sequence and transcriptional regulation. Mol. Endocrinol. 1993, 7:1049-1060.
8. Hong S., Hwang D.Y., Yoon S., Isacson O., Ramezani A., Hawley R.G., Kim K.S. Functional analysis of various promoters in lentiviral vectors at different stages of in vitro differentiation of mouse embryonic stem cells. Mol. Ther. 2007, 15:1630-1639.
9. Kankkonen H.M., Vähäkangas E., Marr R.A., Pakkanen T., Laurema A., Leppänen P., Jalkanen J., Verma I.M., Ylä-Herttuala S. Long-term lowering of plasma cholesterol levels in LDL-receptor-deficient WHHL rabbits by gene therapy. Mol. Ther. 2004, 9:548-556.
10. Kornegay J.N., Li J., Bogan J.R., Bogan D.J., Chen C., Zheng H., Wang B., Qiao C., Howard J.F., Xiao X. Widespread muscle expression of an AAV9 human mini-dystrophin vector after intravenous injection in neonatal dystrophin-deficient dogs. Mol. Ther. 2010, 18:1501-1508.
11. Liew C.G., Draper J.S., Walsh J., Moore H., Andrews P.W. Transient and stable transgene expression in human embryonic stem cells. Stem Cells. 2007, 25:1521-1528.
12. Limberis M.P., Vandenberghe L.H., Zhang L., Pickles R.J., Wilson J.M. Transduction efficiencies of novel AAV vectors in mouse airway epithelium in vivo and human ciliated airway epithelium in vitro. Mol. Ther. 2009, 17:294-301.
13. Londrigan S.L., Brady J.L., Sutherland R.M., Hawthorne W.J., Thomas H.E., Jhala G., Cowan P.J., Kay T.W., O'Connell P.J., Lew A.M. Evaluation of promoters for driving efficient transgene expression in neonatal porcine islets. Xenotransplantation 2007, 14:119-125.
14. Nayak S., Herzog R.W. Progress and prospects: immune responses to viral vectors. Gene Ther. 2010, 17:295-304.
15. Pauwels K., Gijsbers R., Toelen J., Schambach A., Willard-Gallo K., Verheust C., Debyser Z., Herman P. State-of-the-art lentiviral vectors for research use: risk assessment and biosafety recommendations. Curr. Gene Ther. 2009, 9:459-474.
16. Qin J.Y., Zhang L., Clift K.L., Hulur I., Xiang A.P., Ren B.Z., Lahn B.T. Systematic comparison of constitutive promoters and the doxycycline-inducible promoter. PLoS One 2010, 5:e10611.
17. Stoller J.K., Aboussouan L.S. Alpha1-antitrypsin deficiency. Lancet 2005, 365:2225-2236.
18. Wilson A.A., Murphy G.J., Hamakawa H., Kwok L.W., Srinivasan S., Hovav A.H., Mulligan R.C., Amar S., Suki B., Kotton D.N. Amelioration of emphysema in mice through lentiviral transduction of long-lived pulmonary alveolar macrophages. J. Clin. Invest. 2010, 120:379-389.
19. Wang L., Morizono H., Lin J., Bell P., Jones D., McMenamin D., Yu H., Batshaw M.L., Wilson J.M. Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome. Mol. Genet. Metab. 2012, 105:203-211.
20. Xiong W., Goverdhana S., Sciascia S.A., Candolfi M., Zirger J.M., Barcia C., Curtin J.F., King G.D., Jaita G., Liu C., Kroeger K., Agadjanian H., Medina-Kauwe L., Palmer D., Ng P., Lowenstein P.R., Castro M.G. Regulatable gutless adenovirus vectors sustain inducible transgene expression in the brain in the presence of an immune response against adenoviruses. J. Virol. 2006, 80:27-37.
21. Zito E., Buono M., Pepe S., Settembre C., Annunziata I., Surace E.M., Dierks T., Monti M., Cozzolino M., Pucci P., Ballabio A., Cosma M.P. Sulfatase modifying factor 1 trafficking through the cells: from endoplasmic reticulum to the endoplasmic reticulum. EMBO J. 2007, 26:2443-2453.