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Regeneration through iPS cells

Cell therapy is an extremely viable option for the treatment of damage and diseases, also genetic in nature, of skeletal muscles, but different progenitor cell hitherto used; did not provide unequivocal results. Another important issue to consider is that of rejection which greatly restricts the application of cells derived from heterologous sources. A new acquisition of science has solved both problems through the generation of pluripotent cells, iPS cells from adult human tissue (1.2). It has been shown that iPS cells have the same characteristics of embryonic stem cells but do not incur in ethical limitations that prohibit its use.

The therapeutic potential of iPS cells have been demonstrated in many diseases, including, sickle cell anemia, where the phenotype murine was returned to normal thanks to the transplantation of hematopoietic cells derived from iPS-derived autologous (3). Furthermore, iPS cells were induced to differentiation toward the neuronal phenotype, and made capable of improving the cognitive behavior in some animal models of Parkinson’s disease (4). Add to list other diseases in which the use of iPS cells has been instrumental in improving therapeutic Shwachman-Bodian syndrome-Diamond, Gaucher disease type III, Duchenne and Becker muscular dystrophy, Huntington’s disease, juvenile-onset, diabetes mellitus type 1, Down syndrome, Lesch-Nyhan disease.

To date, several groups have generated human iPS cell lines starting from cells of patients with specific pathologies. Park and coworkers have produced a library of iPS lines derived from patients with diseases such as Huntington’s disease, juvenile diabetes mellitus, Down syndrome, muscular dystrophy and many other (5).

IPS cells were derived from different patients and healthy donors thereby providing a library of tissues for the validation of therapies (drugs and testing gene therapy) in vivo individual-specific, thus eliminating the risk of toxicological treatments on the individual.

For what concerns the possible large-scale applications, to be put on a level of importance is the creation of a platform for the generation of standardized skeletal muscle tissue specific to the patient, for wide-ranging therapeutic investigations. Moreover, the increase of the series will create an accurate computer model for the predictability of the onset of genetic diseases, based on individual genetic mutations known and being validated. These studies demonstrate that the use of therapies based on iPS cells is a possible strategy for the treatment of neuro-degenerative and genetic diseases. However, since the lines used in these experiments are obtained via a viral integration, the long-term safety of these iPS cells remains unknown.

Therefore, the development of a strategy that does not require the use of viruses for the generation of iPS cells will be important and could open the door to treatments evolved to different pathologies in a lasting way. In our case, we use messenger RNA coding genes for embryonic reprogramming process, increasing the bio-safety (6) .

We believe that the economic impact of this project can be placed in a broader perspective of health promotion. The investment required will reduce future costs, in terms of clinical intervention, materials and equipment. Personalized medicine is a model that emphasizes in general the “customization” of care, according to which all decisions and practices are tailored on the individual patient and not groups. The basis of personalized medicine is a systematic use of genetic information on an individual patient to select or optimize preventive care or treatment of the same patient. During the last century, the therapy is built on standards of care based on epidemiological studies of large cohorts. On the other hand, these studies do not take into account the genetic variability of the individuals within a population. Personalized medicine aims at laying the groundwork for identifying differences between individuals and then provide individual therapy. The transition from medical groups to a new treatment strategy based on individual biological phenotypes with genomic approaches, proteomic, metabolomic and epigenomici give rise to a new structure in which care will be transformed on the basis of new discoveries in the field of molecular medicine and molecular pathophysiology of disease. This paradigmatic change will depend on the detailed description of the individual biological variation in connection with environmental factors and lifestyles that influence the development of disease.

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