Cell based therapy for duchenne muscular dystrophy
β Scribed by Andrea Farini; Paola Razini; Silvia Erratico; Yvan Torrente; Mirella Meregalli
- Publisher
- John Wiley and Sons
- Year
- 2009
- Tongue
- English
- Weight
- 159 KB
- Volume
- 221
- Category
- Article
- ISSN
- 0021-9541
No coin nor oath required. For personal study only.
β¦ Synopsis
Abstract
Mutations in the dystrophin gene cause an Xβlinked genetic disorder: Duchenne muscular dystrophy (DMD). Stem cell therapy is an attractive method to treat DMD because a small number of cells are required to obtain a therapeutic effect. Here, we discussed about multiple types of myogenic stem cells and their possible use to treat DMD. The identification of a stem cell population providing efficient muscle regeneration is critical for the progression of cell therapy for DMD. We speculated that the most promising possibility for the treatment of DMD is a combination of different approaches, such as gene and stem cell therapy. J. Cell. Physiol. 221: 526β534, 2009. Β© 2009 WileyβLiss, Inc.
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Dystrophin absence in Duchenne muscular dystrophy (DMD) causes severe muscle degeneration. We describe that, as consequence of fibre damage, specific muscle-miRNAs are released into the bloodstream of DMD patients and their levels correlate with the severity of the disease. The same miRNAs are abund
## To the Editor: In a population, when equilibrium between mutation and selection is reached, a fraction (x) of individuals affected by an X-linked recessive lethal disorder is due to new mutations [Haldane, 19351. Being p, the mutation rate in female germ cells, and v, the mutation rate in male