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Vallese, Denis (2013) In vivo behaviour of human precursors into a dystrophic context. [Ph.D. thesis]

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Abstract (english)

The Duchenne Muscular Dystrophy (DMD) is a lethal recessive X-linked disease caused by mutations in the dystrophin gene, no effective treatment is available up to date. The development of effective therapies requires the use of animal models both dystrophic and immunotolerant for human cells.
In collaboration with James Di Santo at the Pasteur Institute, a new mutant strain of mice was created: the Rag2−Il2rb−Dmd−. This mutant lacks T and B cells, as well as NK cells. It also harbours a mutation in the dystrophin gene.
This study focuses on the morphological and immunohistochemical characterization of the muscle phenotype of the new mutant mouse, its comparison with the most used DMD model (mdx) and its potential use to evaluate the regenerative potential of human myogenic progenitors.
The results obtained in this thesis have shown that the dystrophic phenotype is similar between the two models. Analysis of muscle regeneration has yielded very promising results. The Rag2−Il2rb−Dmd− mouse shows a peak of spontaneous regeneration between 10 and 16 weeks, which is six weeks of intense regeneration.
Results obtained after transplantation of human myoblasts have confirmed that this new model is very suitable for myogenic cells transplantation, which can effectively participate in muscle regeneration. This is very important because allows to study the behaviour of human myogenic stem cells in natural dystrophic context.

Abstract (italian)

La distrofia muscolare di Duchenne (DMD) è una malattia letale recessiva legata al cromosoma X causata da mutazioni nel gene della distrofina, nessun trattamento efficace è disponibile al momento. Lo sviluppo di terapie efficaci richiede l'uso di modelli animali sia distrofici che immunotolleranti per le cellule umane.
In collaborazione con James Di Santo presso l'Istituto Pasteur di Parigi abbiamo sviluppato un nuovo modello di topo mutante: il modello Rag2-Il2rb-Dmd-. Questo mutante manca di cellule T e B, così come cellule NK. Inoltre comprende una mutazione nel gene della distrofina.
Questo studio si concentra sulla caratterizzazione morfologica e immunoistochimica del fenotipo muscolare del nuovo modello murino, il confronto con il modello DMD più usato (mdx) e il suo uso potenziale per valutare il potenziale rigenerativo di progenitori miogenici umani.
I risultati ottenuti in questa tesi hanno dimostrato che il fenotipo distrofico è simile tra i due modelli analizzati. L'analisi della rigenerazione muscolare ha dato risultati molto promettenti. Il topo Rag2-Il2rb-DMD- mostra un picco di rigenerazione spontanea tra le 10 e le 16 settimane di età.
I risultati ottenuti dopo il trapianto di mioblasti umani hanno confermato che questo nuovo modello è molto adatto per il trapianto di cellule umane a potenziale miogenico, in grado di partecipare in modo efficace nella rigenerazione muscolare. Ciò è molto importante perché consente di studiare il comportamento delle cellule staminali miogeniche umane in un contesto naturale di distrofia muscolare.

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EPrint type:Ph.D. thesis
Tutor:Vitiello, Libero and Mouly, Vincent
Data di deposito della tesi:23 May 2013
Anno di Pubblicazione:06 May 2013
Key Words:DMD, cell-therapy, mdx, myogenic stem cells ; muscle regeneration ; muscular dystrophies DMD, terapia cellulare, distrofia muscolare, rigenerazione muscolare
Settori scientifico-disciplinari MIUR:Area 05 - Scienze biologiche > BIO/11 Biologia molecolare
Area 05 - Scienze biologiche > BIO/18 Genetica
Struttura di riferimento:Dipartimenti > Dipartimento di Biologia
Codice ID:6114
Depositato il:14 Oct 2013 12:19
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