• 1
    Crystal RG. The alpha 1-antitrypsin gene and its deficiency states. Trends Genet 1989; 5: 411417.
  • 2
    Song S, Morgan M, Ellis T, Poirier A, Chesnur K, Wang J, et al. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proc Natl Acad Sci U S A 1998; 95: 1438414388.
  • 3
    Song S, Laipis PJ, Berns KI, Flotte TR. Effect of DNA-dependent protein kinase on the molecular fate of of the rAAV2 genome in skeletal muscle. Proc Natl Acad Sci U S A 2001; 98: 40844088.
  • 4
    Song S, Embury J, Laipis PJ, Berns KI, Crawford JM, Flotte TR. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Ther 2001; 8: 12991306.
  • 5
    Zhang D, Wu M, Nelson DE, Pasula R, Martin WJ 2nd. Alpha-1-antitrypsin expression in the lung is increased by airway delivery of genetransfected macrophages. Gene Ther 2003; 10: 21482152.
  • 6
    Ferkol T, Mularo F, Hilliard J, Lodish S, Perales JC, Ziady A, et al. Transfer of the human alpha1-antitrypsin gene into pulmonary macrophages in vivo. Am J Respir Cell Mol Biol 1998; 18: 591601.
  • 7
    Asahara T, Kalka C, Isner JM. Stem cell therapy and gene transfer for regeneration. Gene Ther 2000; 7: 451457.
  • 8
    Bordignon C, Roncarolo MG. Therapeutic applications for hematopoietic stem cell gene transfer. Nat Immunol 2002; 3: 318321.
  • 9
    Srivastava A. Obstacles to human hematopoietic stem cell transduction by recombinant adeno-associated virus 2 vectors. J Cell Biochem Suppl 2002; 38: 3945.
  • 10
    Chamberlain JR, Schwarze U, Wang PR, Hirata RK, Hankenson KD, Pace JM, et al. Gene targeting in stem cells from individuals with osteogenesis imperfecta. Science 2004; 303: 11981201.
  • 11
    Xu L, Daly T, Gao C, Flotte TR, Song S, Byrne BJ, et al. CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice. Hum Gene Ther 2001; 12: 563573.
  • 12
    Chao H, Liu Y, Rabinowitz J, Li C, Samulski RJ, Walsh CE. Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther 2000; 2: 619623.
  • 13
    Petersen B, Grossbard B, Hatch H, Pi L, Deng J, Scott EW. Mouse A6 positive hepatic oval cells also express several hematopoietic stem cell markers. Hepatology 2003; In press.
  • 14
    Preisegger KH, Factor VM, Fuchsbichler A, Stumptner C, Denk H, Thorgeirsson SS. Atypical ductular proliferation and its inhibition by transforming growth factor betal in the 3,5-diethoxycarbonyl-1, 4-dihydrocollidine mouse model for chronic alcoholic liver disease. Lab Invest 1999; 79: 103109.
  • 15
    Witek RP, Fisher SH, Petersen BE. Monocrotaline, an alternative to retrorsine based hepatocyte transplantation in rodents. Cell Transplant 2004, in press.
  • 16
    Genin B, Andereggen E, Rubbia-Brandt L, Birraux J, Morel P, Le Coultre C. Improvement of the effect of hepatocyte isograft in the Gunn rat by cotransplantation of islets of Langehans. J Pediatr Surg 1999; 34: 321324.
  • 17
    Oren R, Dabeva MD, Petkov PM, Hurston E, Laconi E, Shafritz DA. Restoration of serum albumin levels in nagase analbuminemic rats by hepatocyte transplantation. Hepatology 1999; 29: 7581.
  • 18
    Schiedner G, Morral N, Parks RJ, Wu Y, Koopmans SC, Langston C, et al. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity [published erratum appears in Nat Genet 1998; 18: 298]. Nat Genet 1998; 18: 180183.
  • 19
    Wilson JM, Johnston DE, Jefferson DM, Mulligan RC. Correction of the genetic defect in hepatocytes from the Watanabe heritable hyperlipidemic rabbit. Proc Natl Acad Sci U S A 1988; 85: 44214425.
  • 20
    Wilson JM, Jefferson DM, Chowdhury JR, Novikoff PM, Johnston DE, Mulligan RC. Retrovirus-mediated transduction of adult hepatocytes. Proc Natl Acad Sci U S A 1988; 85: 30143018.
  • 21
    Kay MA, Li Q, Liu TJ, Leland F, Toman C, Finegold M, et al. Hepatic gene therapy: persistent expression of human alpha 1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther 1992; 3: 641647.
  • 22
    Petersen BE, Bowen WC, Patrene KD, Mars WM, Sullivan AK, Murase N, et al. Bone marrow as a potential source of hepatic oval cells. Science 1999; 284: 11681170.
  • 23
    Ponnazhagan S, Mukherjee P, Wang XS, Qing K, Kube DM, Mah C, et al. Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation. J Virol 1997; 71: 82628267.
  • 24
    Nakai H, Yant SR, Storm TA, Fuess S, Meuse L, Kay MA. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol 2001; 75: 69696976.
  • 25
    Oertel M, Rosencrantz R, Chen YQ, Thota PN, Sandhu JS, Dabeva MD, et al. Repopulation of rat liver by fetal hepatoblasts and adult hepatocytes transduced ex vivo with lentiviral vectors, Hepatology 2003; 37: 9941005.
  • 26
    Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A 2002; 99: 1185411859.