Publication: Neural stem cell-mediated therapy
for rare brain diseases: perspectives
in the near future for LSDs and MNDs
Authors
Filippis, Lidia de
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Publisher
Murcia: F. Hernández y J.F. Madrid, Universidad de Murcia, Departamento de Biología Celular e Histología
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DOI
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info:eu-repo/semantics/article
Description
Abstract
Lysosomal storage diseases (LSDs) are
genetically inherited disorders affecting most patients in
pediatric age and progressively lead to severe, even
lethal, multiorgan dysfunction and brain
neurodegeneration. Motor neuron diseases (MNDs) or
Amyotrophic Lateral Sclerosis (ALS)-related syndromes
are neurodegenerative disorders occurring in the
majority of cases sporadically and affect adult middleaged
patients. Despite being divergent in most
pathological and physiological hallmarks, both MNDs
and LSDs are characterized by tremendous clinical
heterogeneity due to poor prognosis and variable onset
of the symptoms. Moreover, both LSDs and MNDs are
characterized by the concurrence of multiple
pathogenetic processes, such as the development of
inflammatory and excitotoxic environments.
Furthermore, pharmacological, enzyme or genetic
therapies have proven to be ineffective and no cure is
currently available for the neurodegeneration in either
LSD or ALS affected patients. Recent studies have
identified non-neuronal cell types, such as astrocytes and
microglia, as being involved in non cell-autonomous
effects on MND or LSD progression. These findings
have prompted the use of neural stem cells for the
replacement of non-neuronal cells rather than neuronal
cells, which may result in neuroprotection and
immunomodulation. The choice of an appropriate tissue
source and the establishment of standardized paradigms
to culture human neural stem cells (hNSC) will allow
their use for future clinical trials on both ALS and LSD
affected patients and parallel drug screening studies with
novel breakthroughs in the knowledge of
neurodegenerative diseases.
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