utrophin

The unique aspect of today's findings compared to previous studies of exon skipping in mice is that the mice these researchers treated were missing not only dystrophin but another protein known as utrophin, making their disease much more severe and closer to human DMD than the dystrophin-deficient mice previously tested.

Health News from Medical News Today 2009

Note the absence of expression of dystrophin (with the exception of occasional revertant fibers), redistribution of utrophin to the sarcolemma in both regenerating and non-regenerating fibers, expression of developmental myosin heavy chain and reduced/variable expression of β-dystroglycan in the affected dog in comparison with control.

PLoS ONE Alerts: New Articles Gemma L. Walmsley et al. 2010

Researchers have therefore increased the severity of the murine dystrophin-deficiency phenotype by creating double mutants: either mice lacking both dystrophin and its paralogue, utrophin, or both dystrophin and MyoD (a transcription factor important in muscle development and regeneration) [18], [26].

PLoS ONE Alerts: New Articles Gemma L. Walmsley et al. 2010

(Dys1) and carboxy (Dys2) domains, utrophin, developmental myosin heavy chain (dMHC) and β-dystroglycan (bDGC).

PLoS ONE Alerts: New Articles Gemma L. Walmsley et al. 2010

Cerletti M, Negri T, Cozzi F, Colpo R, Andreetta F, et al. (2003) Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer.

PLoS ONE Alerts: New Articles Gemma L. Walmsley et al. 2010

Tinsley JM, Potter AC, Deconinck AE, Davies KE (1998) Skeletal muscle-specific expression of a utrophin transgene rescues utrophin-dystrophin deficient mice.

PLoS ONE Alerts: New Articles Gemma L. Walmsley et al. 2010

The utrophin and developmental myosin heavy chain labeling is on serial sections.

PLoS ONE Alerts: New Articles Gemma L. Walmsley et al. 2010

Working below the DNA level, she is attempting to restart the gene to produce utrophin.

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The idea is to splice RNA to the side that turns off utrophin production and, in theory, let the cell return to making this substance which slows the muscle decline in muscular dystrophy patients.

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(Humans with DMD lack only dystrophin, but for unknown reasons, their disease is more severe than that of mice that lack only dystrophin.) "Here, we report a remarkable prevention of the dystrophic pathology and improvement of the muscle function of the severely affected [dystrophin - and utrophin-deficient] mice," according to study authors.

Health News from Medical News Today 2009