Recombinant adeno-associated virus for muscle directed gene therapy (original) (raw)

• Kotin, R.M. Prospects for the use of adeno-associated virus as a vector for human gene therapy. Hum. Gene Ther. 5, 793–801 (1994).
  Article CAS PubMed Google Scholar
• Kotin, R. et al. Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87, 2211–2215 (1990).
  Article CAS PubMed PubMed Central Google Scholar
• Samulski, R. et al. Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J. 10, 3941–3950 (1991).
  Article CAS PubMed PubMed Central Google Scholar
• Berns, K.I. Parvovirus replication. Microbiol. Rev. 54, 316–329 (1990).
  CAS PubMed PubMed Central Google Scholar
• Samulski, R., Chang, L. & Shenk, T. Helper-free stocks of recombinant adeno-associated viruses: Normal integration does not require viral gene expression. J. Virol. 63, 3822–3828 (1989).
  CAS PubMed PubMed Central Google Scholar
• Russell, D., Miller, A. & Alexander, I. Adeno-associated virus vectors preferentially transduce cells in S phase. Proc. Natl. Acad. Sci. USA 91, 8915–8919 (1994).
  Article CAS PubMed PubMed Central Google Scholar
• Alexander, I.E., Russell, D.W. & Miller, A.D. DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J. Virol. 68, 8282–8287 (1994).
  CAS PubMed PubMed Central Google Scholar
• Russell, D.W., Alexander, I.E. & Miller, A.D. DNA synthesis and topoisomerase inhibitors increase transduction by adeno-associated virus vectors. Proc. Natl. Acad. Sci. USA 92, 5719–5723 (1995).
  Article CAS PubMed PubMed Central Google Scholar
• Fisher, K.J. et al. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70, 520–532 (1996).
  CAS PubMed PubMed Central Google Scholar
• Ferrari, F.K., Samulski, T., Shenk, T. & Samulski, R.J. Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70, 3227–3234 (1996).
  CAS PubMed PubMed Central Google Scholar
• Kumar, S. & Leffak, M. Conserved chromatin structure in c-myc 5′-flanking DNA after viral transduction. J. Mol. Biol. 222, 45–57 (1991).
  Article CAS PubMed Google Scholar
• Kaplitt, M.G. et al. Long-term gene expression and phenotypic correction using adenoassociated virus vectors in the mammalian brain. Nature Genet. 8, 148–154 (1994).
  Article CAS PubMed Google Scholar
• Miller, J.L. et al. Recombinant adeno-associated virus (rAAV)-mediated expression of a human g-globin gene in human progenitor-derived erythroid cells. Proc. Natl. Acad. Sci. USA 91, 10183–10187 (1994).
  Article CAS PubMed PubMed Central Google Scholar
• Podsakoff G., Wong K.K., Jr. & Chatterjee, S. Efficient gene transfer into nondividing cells by adeno-associated virus-based vectors. J. Virol. 68, 5656–5666 (1994).
  CAS PubMed PubMed Central Google Scholar
• Walsh, C.E. et al. Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J. Clin. Invest. 94, 1440–1448 (1994).
  Article CAS PubMed PubMed Central Google Scholar
• Flotte, T.R. et al. Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc. Natl. Acad. Sci. USA 90, 10613–10617 (1993).
  Article CAS PubMed PubMed Central Google Scholar
• Tratschin, J.-D., Miller, I.L., Smith, M.G. & Carter, B.J. Adeno-associated virus vector for high-frequency integration, expression, and rescue of genes in mammalian cells. Mol. Cell. Biol. 5, 3251–3260 (1985).
  Article CAS PubMed PubMed Central Google Scholar
• Muzcyzka, N. Use of adeno-associated virus as a general transduction vector in mammalian cells. Curr. TopicsMicrobiol. Immunol. 158, 97–129 (1992).
  Google Scholar
• McLaughlin, S.K., Collis, P., Hermonat, P.L. & Muzyczka, N. Adeno-associated virus general transduction vectors: Analysis of proviral structures. J. Virol. 62, 1963–1973 (1988).
  CAS PubMed PubMed Central Google Scholar
• Yang, Y., Haecker, S.E., Su, Q. & Wilson, J.M. Immunology of gene therapy with adenoviral vectors in the mouse skeletal muscle. Hum. Molec. Genet. 5, 1703–1712 (1996).
  Article CAS PubMed Google Scholar
• Trask, B.J. Fluorescence in situ hybridization applications in cytogenesis and genemapping. Trends Genet. 7, 149–154 (1991).
  Article CAS PubMed Google Scholar
• Blau, H.M., Pavlath, G.K., Rich, K. & Webster, S.G. Localization of muscle gene products in nuclear domains: Does this constitute a problem for myoblast therapy? Adv. Exp. Med. Biol. 280, 167–172 (1990).
  Article CAS PubMed Google Scholar
• Howe, J.R., Skryabin, B.V., Belcher, S.M., Zerillo, C.A. & Schmauss, C. The responsiveness of a tetracycline-sensitive expression system differs in different cell lines. J. Biol. Chem. 270, 14168–14174 (1995).
  Article CAS PubMed Google Scholar
• Rivera, V.M. et al. A humanized system for pharmacologic control of gene expression. Nature Med. 2, 1028–1032 (1996).
  Article CAS PubMed Google Scholar
• Wolff, J.A. et al. Direct gene transfer into mouse muscle in vivo. Science 247, 1465–1468 (1990).
  Article CAS PubMed Google Scholar
• Tripathy, S.K., Black, H.B., Goldwasser, E. & Leiden, J.M. Immune responses to transgeneencoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med. 2, 545–550 (1996).
  Article CAS PubMed Google Scholar
• Dhawan, J. et al. Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254, 1509–1512 (1991).
  Article CAS PubMed Google Scholar
• Barr, E. & Leiden, J.M. Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507–1509 (1991).
  Article CAS PubMed Google Scholar
• Yao, S.-N., Smith, K.J. & Kurachi, K. Primary myoblast-mediated gene transfer: Persisitent expression of human factor IX in mice. Gene Ther. 1, 99–107 (1994).
  CAS PubMed Google Scholar
• Gussoni, E. et al. A method to co-detect introduced genes and their products in gene therapy protocols. Nature Biotech. 14, 1012–1016 (1996).
  Article CAS Google Scholar
• Bradford, M.M. A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal. Biochem. 72, 248–254 (1976).
  Article CAS PubMed Google Scholar
• Rice, S.A. & Klessig, D.F. Isolation and analysis of adenovirus type 5 mutants containing deletions in the gene encoding the DNA binding protein. J. Virol. 56, 767–778 (1985).
  CAS PubMed PubMed Central Google Scholar
• Wolfe, J.H. et al. Restoration of normal lysosomal function in mucopolysaccharidosis type VII cells by retroviral vector-mediated gene transfer. Proc. Natl. Acad. Sci. USA 87, 2877–81 (1990).
  Article CAS PubMed PubMed Central Google Scholar