Overexpression Of Dystrophin In Transgenic Mdx Mice Eliminates Dystrophic Symptoms Without Toxicity
dc.contributor.author | Cox, G. A. | en_US |
dc.contributor.author | Cole, N. M. | en_US |
dc.contributor.author | Matsumura, K. | en_US |
dc.contributor.author | Phelps, S. F. | en_US |
dc.contributor.author | Hauschka, S. D. | en_US |
dc.contributor.author | Campbell, Kevin P. | en_US |
dc.contributor.author | Faulkner, J. A. | en_US |
dc.contributor.author | Chamberlain, Jeffrey S. | en_US |
dc.date.accessioned | 2009-06-01T17:26:52Z | |
dc.date.available | 2009-06-01T17:26:52Z | |
dc.date.issued | 1993-08-19 | en_US |
dc.identifier.citation | Cox, GA; Cole, NM; Matsumura, K; Phelps, SF; Hauschka, SD; Campbell, KP; Faulkner, JA; Chamberlain, JS. (1993) "Overexpression Of Dystrophin In Transgenic Mdx Mice Eliminates Dystrophic Symptoms Without Toxicity." Nature 364(6439): 725-729. <http://hdl.handle.net/2027.42/62615> | en_US |
dc.identifier.issn | 0028-0836 | en_US |
dc.identifier.uri | https://hdl.handle.net/2027.42/62615 | |
dc.identifier.uri | http://www.ncbi.nlm.nih.gov/sites/entrez?cmd=retrieve&db=pubmed&list_uids=8355788&dopt=citation | en_US |
dc.description.abstract | DUCHENNE and Becker muscular dystrophy (DMD and BMD) are X-linked recessive diseases caused by defective expression of dystrophin1,2. The mdx mouse, an animal model for DMD, has a mutation that eliminates expression of the 427K muscle and brain isoforms of dystrophin1,3,4. Although these animals do not display overt muscle weakness or impaired movement, the diaphragm muscle of the mdx mouse is severely affected and shows progressive myofibre degeneration and fibrosis which closely resembles the human disease5,6. Here we explore the feasibility of gene therapy for DMD by examining the potential of a full-length dystrophin transgene to correct dystrophic symptoms in mdx mice. We find that expression of dystrophin in muscles of transgenic mdx mice eliminates the morphological and immunohistological symptoms of muscular dystrophy. In addition, overexpression of dystrophin prevents the development of the abnormal mechanical properties associated with dystrophic muscle without causing deleterious side effects. Our results provide functional evidence for the feasibility of gene therapy for DMD. | en_US |
dc.format.extent | 889895 bytes | |
dc.format.extent | 2489 bytes | |
dc.format.mimetype | application/octet-stream | |
dc.format.mimetype | text/plain | |
dc.publisher | Macmillan Magazines Ltd. | en_US |
dc.source | Nature | en_US |
dc.title | Overexpression Of Dystrophin In Transgenic Mdx Mice Eliminates Dystrophic Symptoms Without Toxicity | en_US |
dc.type | Article | en_US |
dc.subject.hlbtoplevel | Science | en_US |
dc.description.peerreviewed | Peer Reviewed | en_US |
dc.contributor.affiliationum | UNIV MICHIGAN,SCH MED,DEPT HUMAN GENET,ANN ARBOR,MI 48109 | en_US |
dc.contributor.affiliationum | UNIV MICHIGAN,SCH MED,INST GERONTOL,ANN ARBOR,MI 48109 | en_US |
dc.contributor.affiliationum | UNIV MICHIGAN,SCH MED,CTR HUMAN GENOME,ANN ARBOR,MI 48109 | en_US |
dc.contributor.affiliationother | UNIV IOWA,COLL MED,HOWARD HUGHES MED INST,IOWA CITY,IA 52242 | en_US |
dc.contributor.affiliationother | UNIV IOWA,COLL MED,DEPT PHYSIOL & BIOPHYS,IOWA CITY,IA 52242 | en_US |
dc.contributor.affiliationother | UNIV WASHINGTON,DEPT BIOCHEM,SEATTLE,WA 98195 | en_US |
dc.identifier.pmid | 8355788 | en_US |
dc.description.bitstreamurl | http://deepblue.lib.umich.edu/bitstream/2027.42/62615/1/364725a0.pdf | |
dc.identifier.doi | http://dx.doi.org/10.1038/364725a0 | en_US |
dc.identifier.source | Nature | en_US |
dc.owningcollname | Interdisciplinary and Peer-Reviewed |
Files in this item
Remediation of Harmful Language
The University of Michigan Library aims to describe library materials in a way that respects the people and communities who create, use, and are represented in our collections. Report harmful or offensive language in catalog records, finding aids, or elsewhere in our collections anonymously through our metadata feedback form. More information at Remediation of Harmful Language.
Accessibility
If you are unable to use this file in its current format, please select the Contact Us link and we can modify it to make it more accessible to you.