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iPSC-Based Models to Unravel Key Pathogenetic Processes Underlying Motor Neuron
Disease Development
#MMPMID26237595
Faravelli I
; Frattini E
; Ramirez A
; Stuppia G
; Nizzardo M
; Corti S
J Clin Med
2014[Oct]; 3
(4
): 1124-45
PMID26237595
show ga
Motor neuron diseases (MNDs) are neuromuscular disorders affecting rather
exclusively upper motor neurons (UMNs) and/or lower motor neurons (LMNs). The
clinical phenotype is characterized by muscular weakness and atrophy leading to
paralysis and almost invariably death due to respiratory failure. Adult MNDs
include sporadic and familial amyotrophic lateral sclerosis (sALS-fALS), while
the most common infantile MND is represented by spinal muscular atrophy (SMA). No
effective treatment is ccurrently available for MNDs, as for the vast majority of
neurodegenerative disorders, and cures are limited to supportive care and symptom
relief. The lack of a deep understanding of MND pathogenesis accounts for the
difficulties in finding a cure, together with the scarcity of reliable in vitro
models. Recent progresses in stem cell field, in particular in the generation of
induced Pluripotent Stem Cells (iPSCs) has made possible for the first time
obtaining substantial amounts of human cells to recapitulate in vitro some of the
key pathogenetic processes underlying MNDs. In the present review, recently
published studies involving the use of iPSCs to unravel aspects of ALS and SMA
pathogenesis are discussed with an overview of their implications in the process
of finding a cure for these still orphan disorders.
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