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The ability to reprogram fibroblasts into cardiomyocytes has many therapeutic implications. Half of the cells in the heart are fibroblasts, so the ability to call upon this reservoir of cells already in the organ to become beating heart cells has tremendous promise for cardiac regeneration. Introducing the defined factors, or factors that mimic their effect, directly into the heart to create new heart muscle would avoid the need to inject stem cells into the heart and all the obstacles that go along with such cell-based therapies. In this study Deepak Srivastava and colleagues endeavoured to reprogram committed fibroblasts to cardiomyocytes and started with a pool of 14 candidate cardiomyocyte-inducing factors.

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Duchenne muscular dystrophy (DMD) is due to the absence of dystrophin, which is in itself linked to abnormalities in the DMD gene. However, traces of residual dystrophin and revertant muscle fibres can be found in more or less large numbers in muscle biopsies of patients. In this paper, a British team has demonstrated that of 63 biopsies from DMD patients, 47% were revertant fibres, 32% showed residual traces of dystrophin, 15% were revertant fibres and showed traces of residual dystrophin and 36% did not contain any dystrophin.

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Glycogen storage disease type II or Pompe disease is characterized by muscle weakness, cardiomyopathy and frequent respiratory failure. It is due to the lack of an enzyme, alpha-glucosidase (GAA) or acid maltase, responsible for the degradation of glycogen to glucose in cells. This results in excessive accumulation of glycogen in the lysosomes of cells of different organs. Enzyme replacement therapy is currently the main treatment. In this study, Dutch researchers describe the results of a randomized, placebo-controlled Phase III clinical trial conducted by Genzyme. The trial assessed the efficacy of a recombinant human GAA (alglucosidase alfa or Myozyme) for the treatment of late forms of Pompe disease.

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