Prospects of gene therapy for genetic skeletal muscle disease
Tài liệu tham khảo
Karpati, 1993, The potential for gene therapy in muscular dystrophy and other genetic muscle diseases, Muscle & Nerve, 16, 1141, 10.1002/mus.880161102
Karpati, 1994, The principles of gene therapy in Duchenne muscular dystrophy, Clin Invest Med, 17, 499
Emery, 1988, Duchenne Muscular Dystrophy, 1
Tome, 1994, Congenital muscular dystrophy with merosin deficiency, CR Acad Sci Ser III Sci Vie, 317, 351
Kaplan, 1994, Missense mutations in the adhalin gene linked to autosomal recessive muscular dystrophy, Cell, 78, 625, 10.1016/0092-8674(94)90527-4
Di Mauro, 1995, Biochemistry and molecular genetics of human glycogenoses: An overview, Muscle & Nerve, S510
Acsadi, 1995, The principles of gene therapy for genetic diseases of muscle (Duchenne dystrophy), spinal motor neurons (spinal muscular atrophy) and Schwann cells Schwannopathies), Clin Neurosci, 48, 76
Acsadi, 1995, Adenovirusmediated gene transfer into striated muscles, J Mol Med, 3, 579
Slater, 1994, The anatomy and physiology of the motor unit, 3
Walsh, 1994, Cell biology of muscle, 33
Karpati, 1990, Age-related conversion of dystrophin-negative to -positive fibre segments of skeletal but no cardiac muscle fibres in heterozygote mdx mice, J Neuropath Exp Neurol, 49, 96, 10.1097/00005072-199003000-00002
Carpenter, 1979, Duchenne muscular dystrophy: plasma membrane loss initiates muscle cell necrosis unless it is repaired, Brain, 102, 147, 10.1093/brain/102.1.147
Grounds, 1991, Towards understanding skeletal muscle regeneration, Pathol Res Pract, 118, 1, 10.1016/S0344-0338(11)81039-3
Acsadi, 1994, A different efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity, Hum Mol Genet, 3, 579, 10.1093/hmg/3.4.579
Acsadi, 1991, Human dystrophin expression in mdx mice after injection of DNA constructs, Nature, 352, 815, 10.1038/352815a0
Manthorpe, 1993, Gene therapy by intramuscular injection of plasmid DNA: studies on firefly luciferase gene expression in mice, Hum Gene Ther, 4, 419, 10.1089/hum.1993.4.4-419
Yang, 1990, In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment, 87, 9568
Curiel, 1991, Adenovirus enhancement of transferrin-polylysin-mediated gene delivery, 88, 8850
Vincent, 1994, Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene, Nature Genet, 5, 130, 10.1038/ng1093-130
Ragot, 1993, Efficient adenovirusmediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice, Nature, 361, 647, 10.1038/361647a0
Alameddine, 1994, Expression of a recombinant dystrophin in mdx mice using adenovirus vector, Neuromusc Disord, 4, 193, 10.1016/0960-8966(94)90020-5
Acsadi, 1995, Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer, Hum Gene Ther
Stratford-Perricaudet, 1992, Widespread long-term gene transfer to mouse skeletal muscles and heart via an adenovirus vector, J Clin Invest, 90, 626, 10.1172/JCI115902
Dunckley, 1992, Retroviral-mediated transfer of a dystrophin minigene into mdx mouse myoblasts in vitro, FEBS Lett, 296, 128, 10.1016/0014-5793(92)80363-L
Salvatori, 1993, Retroviral vector-mediated gene transfer into human primary myogenic cells lead to expression in muscle fiber in vivo, Hum Gene Ther, 4, 713, 10.1089/hum.1993.4.6-713
Berkner, 1988, Development of adenovirus vectors for the expression of heterologous genes, Bio Tech, 6, 616
Graham, 1992, Adenovirus-based expression vectors and recombinant vaccines, 363
Graham, 1987, Growth of 293 cells in suspension culture, J Gen Virol, 68, 937, 10.1099/0022-1317-68-3-937
Wickham, 1993, Integrins αvβ3 and αvβ5 promote adenovirus internalization but not virus attachment, Cell, 73, 309, 10.1016/0092-8674(93)90231-E
Jani, 1995, Generation and validation of adenoviral recombinants with large size inserts such as a 6.3 kb human dystrophin cDNA, Virus Res Meth
Yang, 1994, Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy, 91, 4407
Yang, 1995, Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses, J Virol, 69, 2004, 10.1128/JVI.69.4.2004-2015.1995
England, 1990, Very mild muscular dystrophy associated with the deletion of 46% of dystrophin, Nature, 343, 180, 10.1038/343180a0
Hauser, 1994, Expression of dystrophin minigenes in the muscles of mdx mice partially prevents the development of dystrophic symptoms, Am J Hum Genet, 55, A47
Cox, 1993, Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity, Nature, 364, 725, 10.1038/364725a0
Klamut, 1990, Molecular and functional analysis of the muscle-specific promoter region of the Duchenne muscular dystrophy gene, Molec Cell Biol, 10, 193, 10.1128/MCB.10.1.193
Huard, 1995, The route of administration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants, Gene Ther, 2, 107
Sicinski, 1989, The molecular basis of muscular dystrophy in the mdx mouse: a point mutation, Science, 244, 1578, 10.1126/science.2662404
Stedman, 1991, The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy, Nature, 352, 536, 10.1038/352536a0
Kornegay, 1988, Muscular dystrophy in a litter of golden retriever dogs, Muscle & Nerve, 11, 1056, 10.1002/mus.880111008
Arahata, 1993, Laminin in animal models for muscular dystrophy: defect of laminin M in skeletal and cardiac muscle and peripheral nerve of the homozygous dystrophic dy/dy mice, 69B, 259
Sunada, 1994, Deficiency of merosin in dystrophic dy mice and genetic linkage of laminin M chain gene to dy locus, JBiol Chem, 269, 13729, 10.1016/S0021-9258(17)36704-2
Angelos, 1995, Myophosphorylase deficiency associated with rhabdomyolysis and exercise intolerance in 6 related Charolais cattle, Muscle & Nerve, 18, 736, 10.1002/mus.880180710
Wolff, 1992, Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle, Hum Mol Genet, 1, 363, 10.1093/hmg/1.6.363
Lochmuller, 1994, Emergence of early region 1 (E1)-containing replication competent adenovirus in stocks of replication defective adenovirus recombinants (ΔEl+ΔE3) during multiple passages in 293 cells, Hum Gene Ther, 5, 1485, 10.1089/hum.1994.5.12-1485
Danko, 1993, Dystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injection, Hum Mol Genet, 2, 2055, 10.1093/hmg/2.12.2055
Huard, 1995, Differential short-term transduction efficiency of adult versus newborn mouse tissues by adenoviral recombinants, Am J Pathol
Greber, 1993, Stepwise dismantling of adenovirus 2 during entry into cells, Cell, 75, 477, 10.1016/0092-8674(93)90382-Z
Crystal, 1994, Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis, Nature Genet, 8, 42, 10.1038/ng0994-42
Morgan, 1994, Cell and gene therapy in Duchenne muscular dystrophy, Hum Gene Ther, 5, 165, 10.1089/hum.1994.5.2-165
Medical Research Council of Canada, 1990
1993, 921