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It is caused by the mutation or deletion of the <jats:italic>survival motor neuron 1<\/jats:italic> (<jats:italic>SMN1<\/jats:italic>) gene resulting in lower motor neuron (MN) degeneration followed by motor impairment, progressive skeletal muscle paralysis and respiratory failure. In addition to the already existing therapies, a possible combinatorial strategy could be represented by the use of adipose-derived mesenchymal stem cells (ASCs) that can be obtained easily and in large amounts from adipose tissue. Their efficacy seems to be correlated to their paracrine activity and the production of soluble factors released through extracellular vesicles (EVs). EVs are important mediators of intercellular communication with a diameter between 30 and 100\u00a0nm. Their use in other neurodegenerative disorders showed a neuroprotective effect thanks to the release of their content, especially proteins, miRNAs and mRNAs.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Methods<\/jats:title>\n                <jats:p>In this study, we evaluated the effect of EVs isolated from ASCs (ASC-EVs) in the SMN\u03947 mice, a severe SMA model. With this purpose, we performed two administrations of ASC-EVs (0.5\u00a0\u00b5g) in SMA pups via intracerebroventricular injections at post-natal day 3 (P3) and P6. We then assessed the treatment efficacy by behavioural test from P2 to P10 and histological analyses at P10.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Results<\/jats:title>\n                <jats:p>The results showed positive effects of ASC-EVs on the disease progression, with improved motor performance and a significant delay in spinal MN degeneration of treated animals. ASC-EVs could also reduce the apoptotic activation (cleaved Caspase-3) and modulate the neuroinflammation with an observed decreased glial activation in lumbar spinal cord, while at peripheral level ASC-EVs could only partially limit the muscular atrophy and fiber denervation.<\/jats:p>\n              <\/jats:sec><jats:sec>\n                <jats:title>Conclusions<\/jats:title>\n                <jats:p>Our results could encourage the use of ASC-EVs as a therapeutic combinatorial treatment for SMA, bypassing the controversial use of stem cells.<\/jats:p>\n              <\/jats:sec>","DOI":"10.1186\/s13287-024-03693-5","type":"journal-article","created":{"date-parts":[[2024,4,1]],"date-time":"2024-04-01T15:01:52Z","timestamp":1711983712000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Administration of adipose-derived stem cells extracellular vesicles in a murine model of spinal muscular atrophy: effects of a new potential therapeutic strategy"],"prefix":"10.1186","volume":"15","author":[{"given":"Federica","family":"Virla","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ermanna","family":"Turano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Scambi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lorenzo","family":"Schiaffino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marina","family":"Boido","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7392-6689","authenticated-orcid":false,"given":"Raffaella","family":"Mariotti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,4,1]]},"reference":[{"issue":"1","key":"3693_CR1","doi-asserted-by":"publisher","first-page":"124","DOI":"10.1186\/s13023-017-0671-8","volume":"12","author":"IEC Verhaart","year":"2017","unstructured":"Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, et al. 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All mouse experiments used to isolate ASCs were carried out in strict accordance with experimental guidelines approved by the University of Verona committee on animal research (Centro Interdipartimentale di Servizio alla Ricerca Sperimentale) and by the Italian Ministry of Health (protocol #642\/2021-PR, title of approved project \u201cNew therapeutical approach for ALS\u201d). The experimental procedures involving live SMN\u03947 animals were performed in strict accordance with institutional guidelines in compliance with national (D.L. N.26, 04\/03\/2014) and international law and policies (new directive 2010\/63\/EU). The study was approved by the Italian Ministry of Health (protocol #980\/2020-PR, title \u201cEvaluation of the effectiveness of SMN-dependent and SMN-independent therapies to counteract Spinal Muscular Atrophy\u201d). Additionally, the ad hoc Ethical Committee of the University of Turin approved this study. All mice were sacrificed by cervical dislocation or transcardically perfused after being deeply anesthetized by gaseous anaesthesia (See  section). All methods were completed and reported in accordance with the ARRIVE guidelines. The research complies with the commonly accepted \u201c3Rs\u201d, minimizing the number of animals used and avoiding their suffering.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"The authors declare that they have no competing interests.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}}],"article-number":"94"}}