A small regulatory element from chromosome 19 enhances liver-specific gene expression (original) (raw)

• Li C, Bowles DE, van Dyke T, Samulski RJ . Adeno-associated virus vectors: potential applications for cancer gene therapy. Cancer Gene Ther 2005; 12: 913–925.
  Article CAS PubMed PubMed Central Google Scholar
• Warrington Jr KH, Herzog RW . Treatment of human disease by adeno-associated viral gene transfer. Hum Genet 2006; 119: 571–603.
  Article CAS PubMed Google Scholar
• Fu H, Muenzer J, Samulski RJ, Breese G, Sifford J, Zeng X et al. Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brain. Mol Ther 2003; 8: 911–917.
  Article CAS PubMed Google Scholar
• McCarty DM, Fu H, Monahan PE, Toulson CE, Naik P, Samulski RJ . Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Therapy 2003; 10: 2112–2118.
  Article CAS PubMed Google Scholar
• McCarty DM, Monahan PE, Samulski RJ . Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis. Gene Therapy 2001; 8: 1248–1254.
  Article CAS PubMed Google Scholar
• Wang Z, Ma HI, Li J, Sun L, Zhang J, Xiao X . Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Therapy 2003; 10: 2105–2111.
  Article CAS PubMed Google Scholar
• Kotin RM, Linden RM, Berns KI . Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination. EMBO J 1992; 11: 5071–5078.
  Article CAS PubMed PubMed Central Google Scholar
• Lamartina S, Sporeno E, Fattori E, Toniatti C . Characteristics of the adeno-associated virus preintegration site in human chromosome 19: open chromatin conformation and transcription-competent environment. J Virol 2000; 74: 7671–7677.
  Article CAS PubMed PubMed Central Google Scholar
• Ogata T, Kozuka T, Kanda T . Identification of an insulator in AAVS1, a preferred region for integration of adeno-associated virus DNA. J Virol 2003; 77: 9000–9007.
  Article CAS PubMed PubMed Central Google Scholar
• Tan I, Ng CH, Lim L, Leung T . Phosphorylation of a novel myosin binding subunit of protein phosphatase 1 reveals a conserved mechanism in the regulation of actin cytoskeleton. J Biol Chem 2001; 276: 21209–21216.
  Article CAS PubMed Google Scholar
• Flotte TR, Solow R, Owens RA, Afione S, Zeitlin PL, Carter BJ . Gene expression from adeno-associated virus vectors in airway epithelial cells. Am J Respir Cell Mol Biol 1992; 7: 349–356.
  Article CAS PubMed Google Scholar
• Guo ZS, Wang LH, Eisensmith RC, Woo SL . Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer. Gene Therapy 1996; 3: 802–810.
  CAS PubMed Google Scholar
• Prosch S, Stein J, Staak K, Liebenthal C, Volk HD, Kruger DH . Inactivation of the very strong HCMV immediate early promoter by DNA CpG methylation in vitro. Biol Chem Hoppe Seyler 1996; 377: 195–201.
  Article CAS PubMed Google Scholar
• Loser P, Jennings GS, Strauss M, Sandig V . Reactivation of the previously silenced cytomegalovirus major immediate-early promoter in the mouse liver: involvement of NFkappaB. J Virol 1998; 72: 180–190.
  CAS PubMed PubMed Central Google Scholar
• Costa RH, Lai E, Darnell Jr JE . Transcriptional control of the mouse prealbumin (transthyretin) gene: both promoter sequences and a distinct enhancer are cell specific. Mol Cell Biol 1986; 6: 4697–4708.
  Article CAS PubMed PubMed Central Google Scholar
• Bartlett JS, Sethna M, Ramamurthy L, Gowen SA, Samulski RJ, Marzluff WF . Efficient expression of protein coding genes from the murine U1 small nuclear RNA promoters. Proc Natl Acad Sci USA 1996; 93: 8852–8857.
  Article CAS PubMed PubMed Central Google Scholar
• Wu Z, Sun J, Zhang T, Yin C, Yin F, Van Dyke T et al. Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of Hemophilia B at low vector dose. Mol Ther 2007; 16: 280–289.
  Article PubMed Google Scholar
• Wang Z, Zhu T, Qiao C, Zhou L, Wang B, Zhang J et al. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol 2005; 23: 321–328.
  Article CAS PubMed Google Scholar
• Sun B, Zhang H, Franco LM, Young SP, Schneider A, Bird A et al. Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. Mol Ther 2005; 11: 57–65.
  Article CAS PubMed Google Scholar
• Larsen F, Gundersen G, Lopez R, Prydz H . CpG islands as gene markers in the human genome. Genomics 1992; 13: 1095–1107.
  Article CAS PubMed Google Scholar
• Ponger L, Duret L, Mouchiroud D . Determinants of CpG islands: expression in early embryo and isochore structure. Genome Res 2001; 11: 1854–1860.
  Article CAS PubMed PubMed Central Google Scholar
• Yamashita R, Suzuki Y, Sugano S, Nakai K . Genome-wide analysis reveals strong correlation between CpG islands with nearby transcription start sites of genes and their tissue specificity. Gene 2005; 350: 129–136.
  Article CAS PubMed Google Scholar
• Gaszner M, Felsenfeld G . Insulators: exploiting transcriptional and epigenetic mechanisms. Nat Rev Genet 2006; 7: 703–713.
  Article CAS PubMed Google Scholar
• Xiao X, Li J, Samulski RJ . Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224–2232.
  CAS PubMed PubMed Central Google Scholar
• Li C, Hirsch M, Asokan A, Zeithaml B, Ma H, Kafri T et al. Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo. J Virol 2007; 81: 7540–7547.
  Article CAS PubMed PubMed Central Google Scholar