Duchenne Muscular Dystrophy Summary

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Critique of a Possible Gene Therapy for Duchenne Muscular Dystrophy

The article “Rescue of dystrophic skeletal muscle by PGC-1α involves restored expression of dystrophin-associated protein complex components and satellite cell signaling” by Hollinger and others (2013) looked to investigate the effectiveness of Peroxisome Proliferator-activated Receptor Gamma Coactivate 1-alpha (PGC-1α) gene transfer therapy used to alleviate muscle degeneration in people with Duchenne muscular dystrophy (DMD) disease, who essentially lacks protein dystrophin. The authors investigated how PGC-1α would be able to restore the level of dystrophin by upregulating the expression of utrophin, a similar protein, to act as a substitute and prevent the continuing decline of muscle function. Treatment was done on mouse models, then measured utrophin level histologically, PGC-1α expression biochemically, and muscle force functionally. Positive results were mostly interpreted from the data, showing an increase in PGC-1α expression, higher levels of utrophin and associated molecules, and produced stronger contractions in treated muscle cells compared to the controls. This study was well-produced but adjustments and elaboration were needed.

The introduction of this article provided a clear area
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The results fitted nicely with the author’s hypothesis, showing PGC-1α gene transfer was able to control and enhance dystrophin-deficient muscle functioning. Despite successful results, the authors have noted contradicting findings such as increased inflammation in the treated limbs. Little explanation was given as to why and whether it could affect the muscle in the long run, so more elaboration is needed. Overall, the study was well-designed with results to support the hypothesis of this potential treatment for DMD to mitigate symptoms and restore muscle
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