The Current State and Prospects of the Gene Therapy of Duchenne Muscular Dystrophy Worldwide and in Russia

Russian Journal of Genetics

Volumn 37, Issue 8, 2001, Pages 868-875

  Baranov, V S ^a   Baranov, A N ^a   Zelenin, A V ^b  

^a D O OTT RESEARCH INSTITUTE OF OBSTETRICS   (Russian Federation)

^b ENGELHARDT INSTITUTE OF MOLECULAR BIOLOGY   (Russian Federation)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 0345859375     PISSN: 10227954     EISSN: None     Source Type: Journal    
DOI: 10.1023/A:1016765313810     Document Type: Review

References (56)

1. Koenig, M , Hoffman E P , Bertelson, C J , et al , Complete Cloning of the Duchenne Muscular Dystrophy (DMD) cDNA and Preliminary Genomic Organization of the DMD Gene in Normal and Affected Individuals, Cell (Cambridge, Mass), 1987, vol 50, pp 509-517
2. Hoffman, E P , Brown, R H, Jr and Kunkel, L M , Dystiophin The Protein Product of the Duchenne Musculat Dystrophy Locus, Cell (Cambridge, Mass) 1987, vol 51, pp 919-928
3. Den Dunnen J T , Grootscholten, P M , Bakker, E , et al , Topography of the Duchenne Musculai Dystrophy (DMD) Gene FIGE and cDNA Analysis of 194 Cases Reveals 115 Deletions and 13 Duplications, Am J Hum Genet , 1989 vol 45 pp 835-847
4. Roberts, R G , Cofley A J Bobrow, M , et al , Determination of the Exon Structure of the Distal Portion of the Dystrophin Gene by Vectored PCR, Genomics, 1992, vol 13, pp 942-950
5. Partridge T , Dystrophin Gene, Protein, and Cell Biology, Brown S C and Lucy, J A , Eds , Cambridge Univ Press 1997, pp 310-311
6. Bulfield G Siller, W G , Wight, P A, et al , X Chromosome-Linked Muscular Dystrophy (mdx) in the Mouse, Proc Natl Acad Sci USA, 1984, vol 81, pp 1189-1192
7. Sicinski P Geng Y Ryder-Cook, A S , Barnard E A et al , The Molecular Basis of Muscular Dystrophy in the mdx Mouse A Point Mutation, Science, 1989, vol 244, pp 1578-1580
8. Araki E Nakamura K , Nakao K , et al , Targeted Disruption ot Exon 52 in the Mouse Dystrophin Gene Induced Muscle Degeneration Similar to That Observed in Duchenne Musculai Dystiophy, Biochem Biophys Res Commun , 1997, vol 238, pp 492-497
9. Deconinck, N , Imsley, J , De Backer, F , et al , Expression of Truncated Utrophin Leads to Major Functional Improvements in Dystrophin-Deficient Muscles of Mice, Nat Med , 1997 vol 11, pp 1216-1221
10. Cooper, B J , Winand, N J , Stedman, H, et al , The Homologue of the Duchenne Locus Is Detective in X-Linked Muscular Dystrophy of Dogs, Nature, 1988, vol 334, pp 154-156
11. Sharp, N J , Kornegay, J N , Van Camp, S D , et al , An Error in Dystrophin mRNA Processing in Golden Retilever Muscular Dystrophy, an Animal Homologue of Duchenne Muscular Dystrophy, Genomics, 1992, vol 13, pp 115-121
12. Schatzbeig, S J , Olby, N J , Breen, M, et al , Molecular Analysis of Spontaneous Dystrophin "Knockout" Dog, Neuromuscular Disord , 1999, vol 9, pp 289-295
13. Law, P K , Bertorini, T E , Goodwin, T G , et al , Dystrophin Production Induced by Myoblast Transfer Therapy in Duchenne Musculai Dystrophy, Lancet, 1990, vol 14, pp 114-115
14. Baranov, V S , A Report on the IV Working Conference on Gene Therapy of Duchenne Dystrophy, Genetika (Moscow), 1999, vol 35, no 12, pp 1724-1726
15. Beauchamp, J R , Morgan, J E , Pagel, C N , et al , Dynamics of Myoblast Transplantation Reveal a Discrete Minority of Precursor Stem-Line Properties as the Myogenic Source, J Cell Biol , 1999, vol 144, pp 1113-1121
16. Smythe, G M , Hodgetts, S I , and Grounds, M D , Immunobiology and the Future of Myoblast Transfer Therapy, Mol Ther , 2000, no 1, pp 304-313
17. Shishkin, S S , Terekhov, S M , Krokhina, T B , et al , Gene Correction by Means of Cell Dystrophy in Duchenne Muscular Dystrophy, Sbornik otchetov Rossuskoi programmy "Genom cheloveka 2000" (Collection of Reports on the Russian Program "Human Genome-2000"), Moscow, 2001, pp 139-140
18. Sergneurin-Venin, S , Bernard, V , Mersset, P A , et al , Transplantation of Normal and DMD Myoblasts Expressing the Telomerase Gene in SCID Mice, Biochem Biophvs Res Commun , 2000, vol 272, pp 362-369
19. Egorov, E E , Cell "Telomerization" and a Means of Gene Therapy, Gennava terapiya - meditsine budushchego (Gene Therapy tor Future Medicine), Zelenin, A V , Ed , VINITI RAS, 2000, pp 130-132
20. Gussoni, E , Soneoka, Y , Strickland, C D , et al , Dystrophin Expression in the mdx Mouse Restored by Stem Cell Transplantation, Nature, 1999, vol 401, pp 390-394
21. Phelps, S F , Hauser, M A , Cole, N M , et al , Expression of Full-Length and Truncated Dystrophin Minigenes in Transgenic mdx Mice, Hum Mol Genet, 1995, no 4 pp 1251-1258
22. England, S B , Nicholson, L V , Johnson, MA, et al , Very Mild Muscular Dystrophy Associated with the Deletion of 46% of Dystrophin, Nature, 1990, vol 343, pp 180-182
23. Wells, D J , Wells, K E , Walsh, F S , et al , Human Dystrophin Expression Corrects the Myopathic Phenotype in Transgenic mdx Mice, Hum Mol Genet, 1992, vol 1, no 1, pp 35-40
24. Cox, G A , Cole, N M , Matsumura, K , et al , Overexpression of Dystrophin in Transgenic mdx Mice Eliminates Dystrophic Symptoms without Toxicity, Nature, 1993, vol 364, pp 725-729
25. Acsadi, G , Dickson, G , Love, D R , et al , Human Dystrophin Expression in mdx Mice after Intramuscular Injection of DNA Constructs, Nature, 1991, vol 352, pp 815-818
26. Ragot, T , Vincent, N , Chafey, P, et al , Efficient Adenovirus-Mediated Transfer of a Human Minidystrophin Gene to Skeletal Muscle of mdx Mice, Nature, 1993, vol 361, pp 647-650
27. Acsadi, G , Massie, B , and Jani, A , Adenovirus-Mediated Gene Transfer into Striated Muscles, J Mol Med , 1995, vol 73, no 4, pp 165-180
28. Clemens, P R , Kochanek, S , Sumada, Y S , et al , In Vivo Muscle Gene Transfer of Full-Length Dystrophin with Adenovirus Vector That Lacks All Viral Genes, Gene Therapy, 1996, vol 3, no 11, pp 965-972
29. Smaglik, P , Gene Therapy Death Investigators Ponder What Went Wrong, Scientist, 1999, vol 12, no 21, p 1
30. Smaglik, P , Gene Therapy Death May Delay New Trials, Scientist, 1999, vol 13, no 22, p 9
31. Wang, D B and Xiao-Xiao Li, J, Adeno-Associated Virus Vector Carrying Human Minidystrophin Genes Effectively Ameliorates Musculai Dystrophy in mdx Mouse Models, Proc Natl Acad Sci USA, 2000, vol 97, pp 13714-13719
32. Bogdanenko, E V , Sviridov, Yu V Moskovtsev, A A , et al , Nonvural Gene Transfer in Gene Therapy, Vopr Med Khim , 2000, vol 46, no 3, pp 226-245
33. Baranov, V S and Baranov, A N, Gene Therapy of Monogenic Hereditary Disorders Duchenne Muscular Dystrophy, Vopr Med Khim, 2000, vol 46, no 3, pp 279-293
34. Zelenin, A V, Kolesnikov, V A , Tarasenko, O A, et al , Bacterial β-Galactosidase and Human Dystrophin Genes Are Expressed in Mouse Skeletal Muscles after Ballistic Transaction, FEBS Lett, 1997, vol 414, pp 319-392
35. Mir, L M , Bureau, M F, Gehl, J , et al , High-Efficiency Gene Transler into Skeletal Muscle Mediated by Electric Pulses, Proc Natl Acad Sci USA, 1999, vol 96, pp 4262-4267
36. Blaese, R M, Optimism Regarding the Use of RNA/DNA Hybrids to Repair Genes at High Efficiency, J Gene Med, 1999, vol 1, no 2, pp 144-147
37. Rando, T A , Disatnik, M H , and Zhou, L Z, Rescue of Dystrophin Expression in mdx Mouse Muscle by RNA/DNA Oligonucleotides, Proc Natl Acad Sci USA, 2000, vol 97, pp 5363-5368
38. Bartlett, R J , Stockinger, S , Denis, M M , et al , In Vivo Targeted Repair of a Point Mutation in the Canine Dystrophin Gene by a Chimeric RNA/DNA Oligonucleotide, Nat Biotechnol, 2000, vol 18, pp 615-622
39. Wilton, S D , Lloyd, F , Catville, K , et al , Specific Removal of the Nonsense Mutation From the mdx Dystrophin mRNA Using Antisense Oligonucleotides Neuromuscular Disord, 1999, vol 9, no 5, pp 330-338
40. Mann, C J , Honeyman, K , Cheng, A J , et al , Antisense-Induced Exon Skipping and Synthesis of Dystrophin in the mdx Mouse, Proc Natl Acad Sci USA, 2001, vol 98, pp 42-47
41. Burton, E A , Tinsley, J M Holzfernd, P J , et al , A Second Promoter Provides an Alternative Target for Therapeutic Up-Regulation of Utrophin in Duchenne Muscular Dystrophy, Proc Natl Acad Sci USA, 1999, vol 96, pp 14025-14030
42. Tinsley, J M , Potter A C , Phelps S R, et al Amelioration of the Dystrophic Phenotype of mdx Mice Using a Truncated Utrophin Transgene, Nature, 1996, vol 384, pp 349-353
43. Clerk, A , Morris G E , Dubowits, V, et al , Dystrophin-Related Protein, Utrophin, in Normal and Dystrophic Human Fetal Skeletal Muscle, Histochem J, 1993, vol 25, pp 554-561
44. Deconinck, N , Tinsley, J , De Backer, F et al , Expression of Tiuncated Utrophin Leads to Major Functional Improvements in Dystrophin-Deficient Muscles of Mice, Nat Med, 1997, vol 3, pp 1216-1221
45. Wakefield, P M , Tinsley J M , Wood, M J , et al Prevention of Dystrophin Phenotype in the Dystrophin/Utrophin-Deficient Muscle Following Adenovirus-Mediated Transfer of a Utrophin Minigene, Gene Ther, 2000, vol 7, pp 201-204
46. Khurana, T S , Rosmann, A G , Shang J , et al , Activation of Utrophin Promoter by Heregulin via the ETS-Related Transcription Factor Complex GA-Binding Protein α/β, Mol Biol Cell, 1999, vol 10, pp 2075-2086
47. Cotbi, N , Libri V , Fanciulli, M , et al , The Aitificial Zinc Finger Coding Gene "Jazz" Binds the Utrophin Promoter and Activates Transcription, Gene Ther, 2000, vol 7, pp 1076-1083
48. Ferret, A , Wells, K E , and Wells, D J , Immune Responses to Dystrophin Implications for Gene Therapy of Duchenne Muscular Dystrophy, Gene Ther, 2000, vol 7, pp 1439-1446
49. Baranov, V S , Zelenin, A , Baranov, A N , et al , Human Dysrtrophin Gene Expression in mdx Muscles after In Vivo Ballistic Transfection, Application of Synthetic Olygopeptide Complexes and Catronic Liposomes, NATO ASI Ser, Subset M 1997, vol 105, pp 219-223
50. Zelenin A V , Tarasenko, O V , Kolesnikov, V M , et al , Expression of the Human Dystrophin Gene in Skeletal Muscles of mdx Mice alter Ballistic Tiansfection, Genetika (Moscow) 1998, vol 34, no 6, pp 730-736
51. Baranov V S , Tarasenko, O V , Baranov, A N, et al , Expression of the Human Dystrophin Gene in Myofibtils of mdx Mice after Transtection with the Help of Liposomes and Synthetic Ohgopeptides, Genetika (Moscow), 1998, vol 34, no 7, pp 876-882
52. Baranov, A , Glazkov, P , Kiselev, A et al Local and Distant Transfection of mdx Muscle Fiheis with Dystrophin and lacZ Genes Delivered in Vivo by Synthetic Microspheres, Gene Ther, 1999, no 6 pp 1406-1414
53. Smogeeva, N A , Bogdanova, M A Aleinikova, T D , et al , Expression of Marker Genes in Muscular Fibers after In Vivo Lactoferrin-Mediated Transtection, Genetika (Moscow) 2000, vol 36, no 6, pp 749-759
54. Baranov, V S , Zelenin, A V , Ostapenko, O V , et al , Specific Featuies of Transfection and Expression of Gene Constructs Containing the Human Dystrophin cDNA with Various Methods of Virus-Mediated Delivery, Sbornik otchetov Rossinkoi programmy "Genom cheloveka 2000" (Collection of Reports on the Russian Program "Human Genome-2000'), Moscow, 2001, pp 81-83
55. Kiselev, A V , Ostapenko, O V , Batanov, A N , et al , Transfection with Genetic Construction Containing Suppressor tRNA Genes Cause Toxic Effects in Vitro, Abstr ESHG Stockholm, Oct 2000
56. Lukascheva L and Evgrafov, O , Expression of β-Galactosidase Gene in Different Organs of mdx Mice after Intravenous Injection of Naked DNA and DNA-Peptide Complexes, Eur J Hum Genet, 2000, vol 8, no 1, p 174