Data di Pubblicazione:
2015
Citazione:
Stem cell-mediated exon skipping of the dystrophin gene by the bystander effect / M. Meregalli, A. Farini, C. Sitzia, C. Beley, P. Razini, L.M. Cassinelli, F. Colleoni, P. Frattini, N. Santo, E. Galbiati, D. Prosperi, A. Tavelli, M. Belicchi, L. Garcia, Y. Torrente. - In: CURRENT GENE THERAPY. - ISSN 1566-5232. - 15:6(2015 Sep 29), pp. 563-571. [10.2174/1566523215666150929111400]
Abstract:
Duchenne muscular dystrophy (DMD) is characterized by the loss of a functional dystrophin
protein; the muscles of DMD patients progressively degenerate as a result of mechanical stress
during contractions, and the condition eventually leads to premature death. By means antisense oligonucleotides
(AONs), it is possible to modulate pre-mRNA splicing eliminating mutated exons and restoring dystrophin
open reading frame. To overcome the hurdles in using AONs for therapeutic interventions, we exerted engineered human
DMD stem cells with a lentivirus, which permanently and efficiently delivered the cloned AONs. Here we describe for the
first time the exosome-mediated release of AONs from engineered human DMD CD133+ stem cells allowing the rescue
of murine dystrophin expression. Finally, upon release, AONs could be internalized by host cells suggesting a potential
role of exosomes acting as vesicular carriers for DMD gene therapy.
Tipologia IRIS:
01 - Articolo su periodico
Elenco autori:
M. Meregalli, A. Farini, C. Sitzia, C. Beley, P. Razini, L.M. Cassinelli, F. Colleoni, P. Frattini, N. Santo, E. Galbiati, D. Prosperi, A. Tavelli, M. Belicchi, L. Garcia, Y. Torrente
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