Cell therapy is one promising approach to correct genetic diseases by contributing to tissue regeneration; stem cells can be isolated from a healthy donor or, when possible from the same patient. In the first case cells will be transplanted under a regime of immune suppression while in the second case, cells will have to be genetically corrected before transplantation in the same patient from which they were derived. The overall objective of our work is the validation of a clinical treatment for patients affected by Duchenne muscular dystrophy. The project does the groundwork for a phase I/II clinical trial consisting of an intramuscular transplantation of autologous CD133+ stem cells after their engineering through a lentiviral vector. The trial is oriented to DMD boys as Duchenne muscular dystrophy is a X-linked disorder characterized by a mutation in dystrophin gene. Efficacy and possible adverse effects have to be evaluated to test whether this approach may represent a first step towards an efficacious therapy for muscular dystrophy. Our previous works indicated that CD133+ stem cells, a recently identified population of progenitor cells, produce functional improvement upon intra-arterial injection in a mouse model of muscular dystrophy. It thus could be possible to focus on this type of stem cell for autologous transplantation in DMD animal models. Recently transplantation of engineered dystrophic canine muscle-derived CD133+ cells gave promising results in Golden Retriever dystrophic dogs, the most reliable animal model that shows a form of dystrophy very similar to and even more severe than DMD. There is increasing concern in the community of patients that the promise of stem cell research to provide new therapies for yet untreatable diseases will not be fulfilled, at least in the time interval that will be relevant for those who are affected now. It is therefore imperative to move stem cell research into rigorously controlled clinical trials. We believe that the impact of this work will be remarkable, not only in case of a clear positive outcome, but also in case of a modest but significant clinical improvement: indeed it will convince the scientific, the medical and, most importantly, the patient community, that things are moving in the right direction, even though progress towards a definitive treatment will necessarily require further work and time.
|Titolo:||Stem cells in muscle diseases|
|Data di pubblicazione:||29-mag-2016|
|Settore Scientifico Disciplinare:||Settore MED/26 - Neurologia|
|Citazione:||Stem cells in muscle diseases / Y. Torrente. ((Intervento presentato al 2. convegno European Academy of Neurology tenutosi a Copenhagen nel 2016.|
|Appare nelle tipologie:||14 - Intervento a convegno non pubblicato|