Angela Gritti, Ph.D. - "Patient-specific iPSCs for cell therapy applications and disease modelling of lysosomal storage diseases"
Apr 11, 2017
from 12:00 AM to 01:30 AM
|Where||Tigem Auditorium "Vesuvius"|
|Contact Name||Alessandro Fraldi|
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The development of effective and safe neural stem cell-based therapies will depend on procedures that yield well-characterized and expandable autologous cell lines suitable for transplantation that could meet stringent safety criteria and bypass ethical concerns. Here, we will present recent published data showing that bona fide neural stem cells generated via somatic cell reprogramming from fibroblasts of patients affected by Metachromatic Leukodystrophy (MLD) - a genetic demyelinating lysosomal storage disease - can be safely and efficiently engineered and may serve as a stable source of therapeutic enzyme to ameliorate pathology upon intracerebral transplantation and oligodendroglial differentiation in a relevant animal model of the disease (Meneghini et al., 2016). In addition, we will present and discuss unpublished data showing that patient-specific iPSCs and their neuronal and glial progeny (with a particular focus on oligodendrocytes) represent a reliable tool to comprehensively model the degenerative mechanisms underlying MLD pathology (Frati et al, in preparation).