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Cited by 146Amyotrophic Lateral Sclerosis ResearchExtracellular vesicles in diseaseNeurogenetic and Muscular Disorders Research

ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS

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Bonafede, Roberta, Turano, Ermanna, Scambi, Ilaria, Busato, Alice, Bontempi, Pietro, Virla, Federica, Schiaffino, Lorenzo, Marzola, Pasquina +2 more·2020·International Journal of Molecular Sciences
DOI 10.3390/ijms21103651PMC PMC7279464
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Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive degeneration of motoneurons. To date, there is no effective treatment available. Exosomes are extracellular vesicles that play important roles in intercellular communication, recapitulating the effect of origin cells. In this study, we tested the potential neuroprotective effect of exosomes isolated from adipose-derived stem cells (ASC-exosomes) on the in vivo model most widely used to study ALS, the human SOD1 gene with a G93A mutation (SOD1(G93A)) mouse. Moreover, we compared the effect of two different routes of exosomes administration, intravenous and intranasal. The effect of exosomes administration on disease progression was monitored by motor tests and analysis of lumbar motoneurons and glial cells, neuromuscular junction, and muscle. Our results demonstrated that repeated administration of ASC-exosomes improved the motor performance; protected lumbar motoneurons, the neuromuscular junction, and muscle; and decreased the glial cells activation in treated SOD1(G93A) mice. Moreover, exosomes have the ability to home to lesioned ALS regions of the animal brain. These data contribute by providing additional knowledge for the promising use of ASC-exosomes as a therapy in human ALS.

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Cited by

Sorted by citations

Authors
Bonafede, R., Turano, E., Scambi, I., Busato, A., Bontempi, P., Virla, F., Schiaffino, L., Marzola, P., Bonetti, B., & Mariotti, R.
Affiliations
  • University of Verona (IT)
  • Azienda Ospedaliera Universitaria Integrata Verona (IT)
Corresponding author
Mariotti, Raffaella
ORCID
Venue
International Journal of Molecular Sciences
Publisher
MDPI AG
Published
2020-05-21
Volume
21
Issue
10
Pages
3651
ISSN
1422-0067 (ISSN-L)
DOI
10.3390/ijms21103651
PMC
PMC7279464
OpenAlex
W3026155598
Cited by
146
References
1
Citations by year
2026: 23 · 2025: 28 · 2024: 32 · 2023: 18 · 2022: 16 · 2021: 26 · 2020: 3
FWCI
6.82
Type
journal article
Language
en
Open access
Licence
Locations
  • International Journal of Molecular Sciences (publishedVersion, cc-by)
  • PubMed Central (publishedVersion, cc-by)
  • PubMed (publishedVersion)
  • Institutional Research Information System University of Turin (University of Turin) (submittedVersion, other-oa)
  • DOAJ (DOAJ: Directory of Open Access Journals) (submittedVersion, cc-by-sa)
Funders
  • AriSLA (FGBR_7/2016)
  • Brain Research Fondation Verona Onlus (Grant 2017)
Topics
  • Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
  • Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Extracellular vesicles in disease
  • Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
Keywords
amyotrophic lateral sclerosis, exosomes, adipose-derived stem cells, SOD1 G93A mouse model, neuroprotection, motor neuron degeneration, neuromuscular junction, glial activation, intranasal administration, intravenous administration, extracellular vesicles, ALS therapy
MeSH terms
Superoxide Dismutase-1, Adipose Tissue, Amyotrophic Lateral Sclerosis, Animals, Cells, Cultured, Mice, Inbred C57BL, Motor Neurons, Movement, Neuromuscular Junction, Mutation, Missense, Mesenchymal Stem Cell Transplantation, Mice, Exosomes

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