Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for Duchenne muscular dystrophy

Human Molecular Genetics

Volumn 9, Issue 17, 2000, Pages 2507-2515

  Ahmad, A ^a   Brinson, M ^a   Hodges, B L ^a   Chamberlain, J S ^b   Amalfitano, A ^a  

^a DUKE UNIVERSITY MEDICAL CENTER   (United States)

^b UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; TETRACYCLINE; TRANSACTIVATOR PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EMBRYO; FEMALE; GENE EXPRESSION REGULATION; GENE INDUCTION; GENE THERAPY; GENE TRANSFER; GENETIC ENGINEERING; MOLECULAR STABILITY; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PROTEIN STABILITY; SARCOLEMMA; TRANSCRIPTION INITIATION; TRANSCRIPTION REGULATION; TRANSGENIC MOUSE;

ANIMALIA; MUS MUSCULUS;

EID: 0034641893     PISSN: 09646906     EISSN: None     Source Type: Journal    
DOI: 10.1093/hmg/9.17.2507     Document Type: Article

References (28)

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5. Fibre regeneration is not persistent in dystrophic (MDX) mouse skeletal muscle
6. The mdx mouse diaphragm reproduces the degenerative changes of duchenne muscular dystrophy
7. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity
8. Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice
9. Forced expression of dystrophin deletion constructs reveals structure-function correlations
10. Expression of human full-length and minidystrophin in transgenic mdx mice: Implications for gene therapy of duchenne muscular dystrophy
11. Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene
12. A differential efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity
13. Efficiency and functional consequences of adenovirus-mediated in vivo gene transfer to normal and dystrophic (mdx) mouse diaphragm
14. Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene
15. Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles
16. Expression of truncated utrophin leads to major functional improvements in dystrophin-deficient muscles of mice
17. Temporal control of gene expression in transgenic mice by a tetracycline-responsive promoter
18. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters
19. Tetracycline-regulated cardiac gene expression in vivo
20. Efficient control of gene expression by single step integration of the tetracycline system in transgenic mice
21. Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (mdx) mice
22. DNA from both high-capacity and first-generation adenoviral vectors remains intact in skeletal muscle
23. Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells
24. A modified tetracycline-regulated system provides autoregulatory, inducible gene expression in cultured cells and transgenic mice
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26. Transgenic mdx mice expressing dystrophin with a deletion in the actin-binding domain display a 'mild becker' phenotype
27. Persistence of an [E1(-), polymerase(-)] adenovirus vector despite transduction of a neoantigen into immune-competent mice
28. Production and characterization of improved adenovirus vectors with the E1, E2b and E3 genes deleted