Gene Therapy for Muscular Dystrophies: Progress and Challenges

키워드

참고문헌 (46)

1. Acsadi G, "Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs", Nature, 352, pp.815~818 (1991)
2. Aihara H, "Gene transfer into muscle by electroporation in vivo", Nat Biotechnol, 16, pp.867~870 (1998)
3. Mir LM, "High-efficiency gene transfer into skeletal muscle mediated by electric pulses", Proc Natl Acad Sci U S A, 96, pp.4262~4267 (1999)
4. Durieux AC, "In vivo gene electrotransfer into skeletal muscle:effects of plasmid DNA on the occurrence and extent of muscle damage", J Gene Med, 6, pp.809~816 (2004)
5. Molnar MJ, "Factors influencing the efficacy,longevity,and safety of electroporation-assisted plasmid-based gene transfer into mouse muscles", Mol Ther, 10, pp.447~455 (2004)
6. Vilquin JT, "Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies", Gene Ther, 8, pp.1097~1107 (2001)
7. Murakami T, "Full-length dystrophin cDNA transfer into skeletal muscle of adult mdx mice by electroporation", Muscle Nerve, 27, pp.237~241 (2003)
8. McMahon JM, "Optimisation of electrotransfer of plasmid into skeletal muscle by pretreatment with hyaluronidase--increased expression with reduced muscle damage", Gene Ther, 8, pp.1264~1270 (2001)
9. Mennuni C, "Hyaluronidase increases electrogene transfer efficiency in skeletal muscle", Hum Gene Ther, 13, pp.355~365 (2002)
10. Budker V, "The efficient expression of intravascularly delivered DNA in rat muscle", Gene Ther, 5, pp.272~276 (1998)
11. Zhang G, "Intraarterial delivery of naked plasmid DNA expressing full-length mouse dystrophin in the mdx mouse model of duchenne muscular dystrophy", Hum Gene Ther, 15, pp.770~782 (2004)
12. Hagstrom JE, "A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs", Mol Ther, 10, pp.386~398 (2004)
13. Romero NB, "Phase I study of dystrophin plasmid-based gene therapy in Duchenne", Becker muscular dystrophy, 2004, pp.15, 1065~1076
14. DelloRusso C, "Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing fulll-ength dystrophin", Proc Natl Acad Sci U S A, 99, pp.12979~12984 (2002)
15. Nunes FA, "Gene transfer into the liver of nonhuman primates with E1-deleted recombinant adenoviral vectors:safety of readministration", Hum Gene Ther, 10, pp.2515~2526 (1999)
16. Jooss K, "Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers", J Virol, 72, pp.4212~4223 (1998)
17. Wang B, "Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model", Proc Natl Acad Sci U S A, 97, pp.13714~13719 (2000)
18. Yoshimura M, "AAV vector-mediated microdystrophin expression in a relatively small percentage of mdx myofibers improved the mdx phenotype", Mol Ther, 10, pp.821~828 (2004)
19. Gregorevic P, "Systemic delivery of genes to striated muscles using adeno-associated viral vectors", Nat Med, 10, pp.828~834 (2004)
20. Rodino-Klapac LR, "Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D", Neurology, 71, pp.240~247 (2008)
21. Yuasa K, "Adeno-associated virus vector-mediated gene transfer into dystrophin-deficient skeletal muscles evokes enhanced immune response against the transgene product", Gene Ther, 9, pp.1576~1588 (2002)
22. Rivière C, "Long-term expression and repeated administration of AAV type 1,2 and 5 vectors in skeletal muscle of immunocompetent adult mice", Gene Ther, 13, pp.1300~1308 (2006)
23. Gregorevic P, "rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice", Nat Med, 12, pp.787~789 (2006)
24. Inagaki K, "Robust systemic transduction with AAV9 vectors in mice:efficient global cardiac gene transfer superior to that of AAV8", Mol Ther, 14, pp.45~53 (2006)
25. Mendell JR, "Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins", Ann Neurol, 66, pp.290~297 (2009)
26. Li J, "rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy", Gene Ther, 6, pp.74~82 (1999)
27. Monaco AP, "An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus", Genomics, 2, pp.90~95 (1988)
28. Dunckley MG, "Modification of splicing in the dystrophin gene in cultured Mdx muscle cells by antisense oligoribonucleotides", Hum Mol Genet, 7, pp.1083~1090 (1998)
29. Mann CJ, "Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse", Proc Natl Acad Sci U S A, 98, pp.42~47 (2001)
30. Alter J, "Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology", Nat Med, 12, pp.175~177 (2006)
31. Lu QL, "Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse", Nat Med, 9, pp.1009~1014 (2003)
32. Wells KE, "Enhanced in vivo delivery of antisense oligonucleotides to restore dystrophin expression in adult mdx mouse muscle", FEBS Lett, 552, pp.145~149 (2003)
33. Bremmer-Bout M, "Targeted exon skipping in transgenic hDMD mice:a model for direct preclinical screening of human-specific antisense oligonucleotides", Mol Ther, 10, pp.232~240 (2004)
34. Takeshima Y, "Intravenous infusion of an antisense oligonucleotide results in exon skipping in muscle dystrophin mRNA of Duchenne muscular dystrophy", Pediatr Res, 59, pp.690~694 (2006)
35. van Deutekom JC, "Local dystrophin restoration with antisense oligonucleotide PRO051", N Engl J Med, 357, pp.2677~2686 (2007)
36. Barton-Davis ER, "Aminoglycoside antibiotics restore dystrophin function to skeletal muscles of mdx mice", J Clin Invest, 104, pp.375~381 (1999)
37. Wagner KR, "Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations", Ann Neurol, 49, pp.706~711 (2001)
38. Politano L, "Gentamicin administration in Duchenne patients with premature stop codon.Preliminary results", Acta Myol, 22, pp.15~21 (2003)
39. Howard MT, "Sequence specificity of aminoglycoside-induced stop condon readthrough:potential implications for treatment of Duchenne muscular dystrophy", Ann Neurol, 48, pp.164~169 (2000)
40. Welch EM, "PTC124 targets genetic disorders caused by nonsense mutations", Nature, 447, pp.87~91 (2007)
41. Hirawat S, "Safety,tolerability,and pharmacokinetics of PTC124,a nonaminoglycoside nonsense mutation suppressor,following single-and multiple-dose administration to healthy male and female adult volunteers", J Clin Pharmacol, 47, pp.430~444 (2007)
42. Quenneville SP, "Dystrophin expression in host muscle following transplantation of muscle precursor cells modified with the phiC31 integrase", Gene Ther, 14, pp.514~522 (2007)
43. Ikemoto M, "Autologous transplantation of SM", C-2.6(+)satellite cells transduced with micro-dystrophin CS1 cDNA by lentiviral vector into mdx mice, 2007, pp.15, 2178~2185
44. Benchaouir R, "Restoration of human dystrophin following transplantation of exon-skipping-engineered DMD patient stem cells into dystrophic mice", Cell Stem Cell, 1, pp.646~657 (2007)
45. Sampaolesi M, "Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs", Nature, 444, pp.574~579 (2006)
46. Torrente Y, "Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients", Cell Transplant, 16, pp.563~577 (2007)

관련 논문AI추천