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ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS
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 effe...
Autores principales: | , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7279464/ https://www.ncbi.nlm.nih.gov/pubmed/32455791 http://dx.doi.org/10.3390/ijms21103651 |
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author | Bonafede, Roberta Turano, Ermanna Scambi, Ilaria Busato, Alice Bontempi, Pietro Virla, Federica Schiaffino, Lorenzo Marzola, Pasquina Bonetti, Bruno Mariotti, Raffaella |
author_facet | Bonafede, Roberta Turano, Ermanna Scambi, Ilaria Busato, Alice Bontempi, Pietro Virla, Federica Schiaffino, Lorenzo Marzola, Pasquina Bonetti, Bruno Mariotti, Raffaella |
author_sort | Bonafede, Roberta |
collection | PubMed |
description | 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. |
format | Online Article Text |
id | pubmed-7279464 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2020 |
publisher | MDPI |
record_format | MEDLINE/PubMed |
spelling | pubmed-72794642020-06-17 ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS Bonafede, Roberta Turano, Ermanna Scambi, Ilaria Busato, Alice Bontempi, Pietro Virla, Federica Schiaffino, Lorenzo Marzola, Pasquina Bonetti, Bruno Mariotti, Raffaella Int J Mol Sci Article 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. MDPI 2020-05-21 /pmc/articles/PMC7279464/ /pubmed/32455791 http://dx.doi.org/10.3390/ijms21103651 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/). |
spellingShingle | Article Bonafede, Roberta Turano, Ermanna Scambi, Ilaria Busato, Alice Bontempi, Pietro Virla, Federica Schiaffino, Lorenzo Marzola, Pasquina Bonetti, Bruno Mariotti, Raffaella ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS |
title | ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS |
title_full | ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS |
title_fullStr | ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS |
title_full_unstemmed | ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS |
title_short | ASC-Exosomes Ameliorate the Disease Progression in SOD1(G93A) Murine Model Underlining Their Potential Therapeutic Use in Human ALS |
title_sort | asc-exosomes ameliorate the disease progression in sod1(g93a) murine model underlining their potential therapeutic use in human als |
topic | Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7279464/ https://www.ncbi.nlm.nih.gov/pubmed/32455791 http://dx.doi.org/10.3390/ijms21103651 |
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