MAURILIO SAMPAOLESI

Full professor

email: maurilio.sampaolesi@uniroma1.it
phone: +39 06 49766754
building: RM008
room: studio 13

Education
1997-1999: Postdoctoral Associate, Molecular Physiology Dept, NCVC, Suita-city, Osaka, Japan.
1997: PhD in “Cardiac Pathophysiology”, University of Tor Vergata, Rome, Italy
1994: National Board Certification, Italy ID # 044660
1991: Bachelor/master’s degree in Biological Sciences, La Sapienza University, Rome, Italy

Position(s)
2021-, Professor of Histology and Medical Embryology, Sapienza University of Rome, Italy
2021-, Head of Stem Cell and Developmental Biology Unit, KU Leuven, Belgium
2018-, Professor of Stem Cell Biology, Department of Development and Regeneration, KU Leuven,
2011-2021- Associate Professor of Human Anatomy, University of Pavia, Italy
2007-, Head of Translational Cardiomyology Laboratory, Leuven, Belgium
2005-20011, Assistant Professor of Human Anatomy, University of Pavia, Italy
2000-2005, PI, Téléthon Foundation, San Raffaele Hospital Milan, Italy
1999-2000, PI, Pharmaceutical Company Dompé SpA, L’Aquila, Italy

Scientific track-record
Maurilio Sampaolesi was initially trained as a biologist at La Sapienza University of Rome Italy and after getting a PhD in Cardiovascular Pathophysiology at Tor Vergata University of Rome, he moved to the National Cardiovascular Center, Osaka, Japan for a postdoctoral fellowship in 1997 in the lab of M. Shigekawa. He joined the Stem Cell Institute (director G. Cossu) in Milan, Italy, in 2000 as senior researcher, after a tenured position as consultant at Dompe` Pharmaceutical Company, Italy. He participated to the identification of vessel associated stem cells in skeletal muscles and named them mesoangioblasts (MABs). MABs have been shown to be effective in restoring function in murine (Sampaolesi et al Science 2003) and canine models (Sampaolesi et al. Nature 2006) of muscular dystrophy. The Sampaolesi research group in the last 10 years has accumulated extensive experience in the field of stem cells and their use for the treatment of skeletal muscle diseases, focusing on Duchenne and limb girdle muscular dystrophies. More specifically the group has been working on the role of miRNAs (Crippa et al. Curr Pharm Des. 2012; Quattrocelli & Sampaolesi Adv Drug Deliv Rev. 2015) in the control of different aspects of cardiac and muscle functions. They reported that mesodermal progenitors from Sgcb-null mice, an animal model for limb girdle muscular dystrophy type 2 E, displaying an aberrant activation of skeletal muscle master genes due to the dysregulation of miR669 family (Crippa et al. JCB 2011). Indeed, the miR669a administration ameliorated Sgcb-null cardiac degeneration (Quattrocelli et al. JAHA 2013). The group is currently focusing on human induced pluripotent stem cells and their derivatives with myogenic differentiation potential (Quattrocelli et al JCI 2015). Basic studies related to the roles of epigenetics (methylation and histone code), miRNAs in myogenic commitments (Giacomazzi et al. Nat Comm 2017; Costamagna et al. J Cachexia Sarcopenia Muscle. 2020; Giarratana et al. Cell Death & Dis. 2020) and single cell studies (Camps et al. Cell Reports 2020; Breuls. et al. Cell Death & Dis. 2021) are also main research tasks. They put effort on basic studies on epigenetic memory (methylation and histone code), miRNA signatures and single cell identity (scRNAseq / scATAC-seq 10x Genomics) towards myogenic lineages. Finally, 2D and 3D skeletal and cardiac cell models (Marini et al. Front Cell Dev Biol. 2022) exosome mediated protein and miRNA cargo networking to force myogenic differentiation in fusion-negative rhabdomyosarcoma cells (Pozzo et al. Front Physiol. 2021) or to induce muscle hypertrophy (Yedigaryan et al. Front Immunol. 2022) are the most recent scientific adventures.

Selected invited presentations (from a total >30 conference presentations since 2012
1. 2D-3D Stem Cell Models for Genetic Myopathies: Exploring Therapeutic Options. 10th Annual Cystinosis Workshop, Dublin, Ireland 9-10 February 2024
2. Stem cells for muscle repair and 3D pathophysiological models. XXXIX Congresso Società Italiana di Istochimica, Vulcano, Italy 14-16 June 2023
3. Harnessing novel 3D stem cell-based models to challenge dystrophic cardiomyopathy. 5th Central European Biomedical Congress, Krakow, Poland 30 May 2023
4. 2D and 3D stem cell-based models of dystrophic cardiomyopathy.34 AICCannual meeting Orgnaoid models fof human diseases. Naples, Italy 2 Dec 2022
5. Generate Your Muscle (GYM): Development of autologous myogenic stem cell therapy to treat myopathy Maastricht 8 Dec 2020
6. Single-cell omics to decomplexify muscle stem cell heterogeneity. University of Geneve 6 Feb 2020
7. Cell reprogramming, gene-editing and single-cell seq technologies. Unistem Milan 6 June 2019
8. Epigenetic memory in skeletal muscle remodelling and regeneration. St. Ann's University, Brno, 20 Feb2018
9. Muscle stem cells and regeneration. Muscle biology & inflammation. EULAR, London 6 June 2016
10. Cell reprogramming and miRNA-based treatments for skeletal and cardiac muscle repair. Enrlangen University, 15 June 2015.
11. Novel routes towards the 'heart' of myogenesis IIIM 2014 - XI meeting Monteriggioni, Italy 2-5 October 2014
12. Cardiac progenitors in dystrophic stem cell niche. 7th Qianjiang International Cardiovascular Congress (QICC) Hangzhou, China 1 August 2013
13. Stem cell treatment for muscular dystrophy: where we are and where we go. NCVC and Osaka University, Suita, Japan 12 June 2012
14. miRNAs regulate muscle lineage switch, NIRM Amsterdam, The Netherlands, 20 September 2011
15. Magic-factor 1 and muscle hypertrophy. 5th Cachexia World Conference Barcellona, Spain, 5-8 December 2009

Fellowships and awards
2018 Awarded the best oral and poster presentation - Premio Prof. Paolo Bianco IX Stem Cell Res, Milano
2011: Awarded the best Poster Presentation, ASCR EMBO Meeting, Paris, France
2008: Awarded the scientific project on cardiac regeneration, FWO-Odysseus Brussels, Belgium 2007: Awarded the best scientific project on Stem Cell Technology, Mai Foundation, Milan, Italy 2006: Awarded the best scientific team at San Raffaele Scientific Institute Retreat, Bardolino, Italy
2000: Grant 463/bi Task Force on “Stem cell therapy for muscular dystrophies” Telethon foundation, Italy 1997: Awarded the best scientific project on Muscular Dystrophy. JISTEC Osaka, Japan

Memberships of scientific societies and editorial activities
Member European Society of Gene and cell Therapy, International Society for Stem Cell Research (ISSCR), Belgian Society for Stem Cell Research (BESSCR). Deputy Editor (2013-2017) and Associate Editor for Cardiovascular Research (CVR). Academic Editor Scientific Reports and International Journal of Molecular Sciences (since2018); Academic Editor for Frontiers Journals, Stem Cell Research Panel (since 2012) Academic Editor for PLOSONE (since 2010). Belgian Society for Stem Cell Research (BESSCR, since2016); European Society of Gene and Cell Therapy (ESCCT, since 2008); International Society for Stem Cell Research (ISSCR since2005). Scientific board: Human Gene Therapy, Cell and Developmental Biology, BAM Basic and Applied Myology. The European Journal of Translational Myology. Reviewer for several Journals, including Biomaterial, Cell Death and Differentiation, FASEB Journal, Human Gene Therapy, PLOSONE, Stem Cells, Trends in Molecular Medicine, Tissue engineering, Journal of Cell Science, Cell Transplantation.

Commissions of trust
Panel member of Stem Cell Committee, AFM-Telethon France since 2012
Panel reviewer ERC starting (2010-) consolidator (2014-) and advanced grants (2018-)
Grant reviewer Clinical and Applied Biomedical Research, Health Research Board of Ireland since 2009 Grant reviewer for the Research Grants Council (RGC) of Hong Kong since 2008

Organisation of scientific meetings
Co-organizer of Interuniversity Institute of Miology meetings (since 2008/annual meeting) IIM Meeting 2023 will be held in Assisi (Perugia Italy), October 23-25, 2021.
Responsible for Stem Cell Research seminars series since 2010 at Department of Development and Regeneration, KU Leuven CARE-MI: multicenter FP7-HEALTH-2009-1.4-3 Large Scale integrating project: Activation of endogenous cells as an approach to regenerative medicine. 12-13 July 2012 Leuven, Belgium. Muscular Dystrophy Symposium October 2, 2008 Leuven, Belgium.

Career breaks
None

Contribution to early careers:
Successfully mentoring 2-4 Master/Erasmus students/year since 2008, 15 postdocs, 25 PhD students defending their PhD thesis within 4 years and occasionally with an extra period that never exceeded 5 years. Young researchers supervised during the academic career as now independent scientists: Stefano Biressi, Associate Professor University of Trento Italy; Lieven Thorrez, Professor, Kulak KU Leuven, Belgium; Marco Cassano, Associate Professor at University of Milan, Flavio Ronzoni, Assistant Professor, Humanitas, Italy others got FWO postdoctoral fellowships (Emanuele Berardi, and Mattia Quattrocelli, currently PoStDoc VIB-KU Leuven, Belgium and Assistant Professor, UC Department of Pediatrics, Cincinnati USA respectively). I am also involved in science days at KU Leuven for children and their parents to be experienced in stem cell research.

Leadership in industrial innovation
Sampaolesi team filed two patent applications: 1) Skeletal muscle differentiation of mesodermal IPSC derived progenitors. UK Patent Application Ref. No GB1717939.1. Filed in the UK October 31st, 2017 (WO 2019086515. PCT/EP2018/079826) P.R. Maurilio Sampaolesi Giorgia Giacomazzi and Enrico Pozzo; 2) Mesodermal progenitor cells. UK Patent Application Ref. No. GB1408712.6. Filed in the UK May 16th, 2014 (WO 2015172207 A1. PCT/BE2015/00002) P.R. Maurilio Sampaolesi and Mattia Quattrocelli. R&D projects with MonaCell bvba, Belgium sponsored by IWT. In addition, Dompe, Biotech (Italy) Coretherapix S.L. (Spain), iTeos therapeutics (Belgium) and Evox therapeutics (United Kingdom) are directly involved in ongoing projects mainly related to miRNA technologies. Finally, Sampaolesi was consortium member and promoter of EU educational grant Erasmus plus, SupBioEnt: Supporting biotechnology students oriented towards an entrepreneurial path together European partners (2017-2019).

Achievements
1. We provided evidence of Areg (Adipogenic regulator cells) in dystrophic muscles by single cell RNAseq and showed that IL4 treatment iwas able to counteract muscle atrophy. Camps J, Breuls N, Sifrim A,…, Voet T, Sampaolesi M. Interstitial Cell Remodeling Promotes Aberrant Adipogenesis in Dystrophic Muscles. Cell Rep. 2020, 31(5):107597 (IF: 8.109). Costamagna D, Duelen R, Penna F, Neumann D, Costelli P, Sampaolesi M. Interleukin-4 administration improves muscle function, adult myogenesis, and lifespan of colon carcinoma-bearing mice. J Cachexia Sarcopenia Muscle. 2020 Jun;11(3):783-801.) (IF: 12.910)

2. Valorization of epigenetic cell memory in iPS derivatives: Giacomazzi G, Holvoet B, Trenson S, Caluwé E, Kravic B, Grosemans H, Cortés-Calabuig Á, Deroose CM, Huylebroeck D, Hashemolhosseini S, Janssens S, McNally E, Quattrocelli M, Sampaolesi M. MicroRNAs promote skeletal muscle differentiation of mesodermal iPSC-derived progenitors. Nat Commun. 2017 8(1):1249. Quattrocelli M…and Sampaolesi M. Mesodermal iPSC-derived progenitor cells functionally regenerate cardiac and skeletal muscle. J Clin Invest. 2015 (recent IF= 13.3). The epigenetic memory was proven also in equine iPS cells: Quattrocelli M, Giacomazzi G, Broeckx SY, Ceelen L, Bolca S, Spaas JH, Sampaolesi M.. Equine-Induced Pluripotent Stem Cells Retain Lineage Commitment Toward Myogenic and Chondrogenic Fates. Stem Cell Reports. 2016 6(1): 55-63. Patents as inventor: Generation of mesodermal iPSC- derived progenitors (WO2015172207, 2015-11-19); muscle differentiation of mesodermal iPSC-derived progenitors using miRNAs (WO2019086515A1, 2019-05-09). The inventions relate to methods for culturing and generation of mesodermal progenitor cells from pluripotent stem cells and to improve myogenesis by miRNA/antagomiR perturbations.

3. Transposon-based ex vivo gene therapy for muscular dystrophy (MD). Loperfido M, Jarmin S, Dastidar S, Di Matteo M, Perini I, Moore M, Nair N, Samara-Kuko E, Athanasopoulos T, Tedesco FS, Dickson G, Sampaolesi M*, VandenDriessche T*, Chuah MK*. piggyBac transposons expressing full-length human dystrophin enable genetic correction of dystrophic mesoangioblasts. Nucleic Acids Res. 2016 44(2): 744-60 *senior authors (IF: 10.162)

4. miRNAs involved in cardiac degeneration linked to muscular dystrophy and miRNA gene therapy. We provided the first evidence of miRNA targeting MyoD and rescue a genetic defect by miRNA treatment. Crippa S, Cassano M, Messina G, Galli D, Galvez BG, Curk T, Altomare C, Ronzoni F, Toelen J, Gijsbers R, Debyser Z, Janssens S, Zupan B, Zaza A, Cossu G, Sampaolesi M. miR669a and miR669q act as cell fate switch between cardiac and skeletal muscle lineages. J Cell Biol 2011; 193(7): 1197-212 (IF:10,264); Quattrocelli M, Crippa S, Montecchiani C, Camps J, Cornaglia AI, Boldrin L, Morgan J, Calligaro A, Casasco A, Orlacchio A, Gijsbers R, D'Hooge J, Toelen J, Janssens S, Sampaolesi M. Long-term miR- 669a therapy alleviates chronic dilated cardiomyopathy in dystrophic mice. J Am Heart Assoc. 2013, 2(4): e000284 (IF: 2,882);

5. Stem cell therapy protocols: from mice to large animal model for MDs and establish pluripotent stem cell technology at the Laboratory of Translational Cardiomyology (2010). After studying adult stem cells to boost myogenesis (Sampaolesi et al. Science 2003, Nature 2006. Nature Cell Biology 2007) we extensively worked on cell reprogramming and published the first evidence of myogenic cell memory in iPS cells: Quattrocelli M, Palazzolo G, Floris G, Schöffski P, Anastasia L, Orlacchio A, Vandendriessche T, Chuah MK, Cossu G, Verfaillie C, Sampaolesi M. Intrinsic cell memory reinforces myogenic commitment of pericyte- derived iPSCs. J Pathol. 2011 Apr;223(5):593-603 (IF: 6.318)

Selected 15 publications
(https://orcid.org/0000-0002-2422-3757; Researcher ID: H-6509-2013; H-index: 50 (Google Scholar, i10-index: 125, >13500 total citations; H-index: 43 (Scopus) >170 ms in PubMed)
1. Vervliet T, Duelen R, Pradhan A, La Rovere R, Roderick HL, Sampaolesi M. Cardiomyocyte differentiation from human induced pluripotent stem cells is delayed following knockout of Bcl-2. J Cell Sci. 2023 136(2): jcs 260216. (recent IF: 5.285)
2. Yedigaryan L, Martínez-Sarrà E, Giacomazzi G, Giarratana N, van der Veer BK, Rotini A, Querceto S, Grosemans H, Cortés-Calabuig Á, Salucci S, Battistelli M, Falcieri E, Gijsbers R, Quattrocelli M, Peng Koh K, De Waele L, Buyse GM, Derua R, Sampaolesi M. Extracellular vesicle-derived miRNAs improve stem cell-based therapeutic approaches in muscle wasting conditions. Front Immunol. 2022 13:977617 (recent IF: 8.786)
3. Marini V, Marino F, Aliberti F, Giarratana N, Pozzo E, Duelen R, Cortés Calabuig Á, La Rovere R, Vervliet T, Torella D, Bultynck G, Sampaolesi M*, Chai YC*. Long-term culture of patient-derived cardiac organoids recapitulated Duchenne muscular dystrophy cardiomyopathy and disease progression. Front Cell Dev Biol. 2022 10:878311 (recent IF: 6.684) *equally contributors
4. Duelen R, Costamagna D, Gilbert G, Waele L, Goemans N, Desloovere K, Verfaillie CM, Sipido KR, Buyse GM, Sampaolesi M. Human iPSC model reveals a central role for NOX4 and oxidative stress in Duchenne cardiomyopathy. Stem Cell Reports. 2022 17(2):352 (recent IF: 7.765)
5. Breuls N, Giarratana N, Yedigaryan L, Garrido GM, Carai P, Heymans S, Ranga A, Deroose C, Sampaolesi M. Valproic acid stimulates myogenesis in pluripotent stem cell-derived mesodermal progenitors in a NOTCH-dependent manner. Cell Death Dis. 2021 12(7):677. (IF: 9.69)
6. Machado L, Geara P, Camps J, Dos Santos M, Teixeira-Clerc F, Van Herck J, Varet H, Legendre R, Pawlotsky JM, Sampaolesi M, Voet T, Maire P, Relaix F, Mourikis P. Tissue damage induces a conserved stress response that initiates quiescent muscle stem cell activation. Cell Stem Cell. 2021 28(6):1125-1135.e7. (IF: 24.66)
7. Costamagna D, Duelen R, Penna F, Neumann D, Costelli P, Sampaolesi M. Interleukin-4 administration improves muscle function, adult myogenesis, and lifespan of colon carcinoma-bearing mice. J Cachexia Sarcopenia Muscle. 2020 11(3):783-801, (IF: 12.91)
8. Giarratana N, Conti F, La Rovere R, Gijsbers R, Carai P, Duelen R, Vervliet T, Bultynck G, Ronzoni F, Piciotti R, Costamagna D, Fulle S, Barravecchia I, Angeloni D, Torrente Y, Sampaolesi M. MICAL2 is essential for myogenic lineage commitment. Cell Death Dis. 2020 11(8):654. (IF: 8.469)
9. Camps J, Breuls N, Sifrim A, Giarratana N, Corvelyn M, Danti L, Grosemans H, Vanuytven S, Thiry I, Belicchi M, Meregalli M, Platko K, MacDonald ME, Austin RC, Gijsbers R, Cossu G, Torrente Y, Voet T, Sampaolesi M. Interstitial Cell Remodeling Promotes Aberrant Adipogenesis in Dystrophic Muscles. Cell Rep. 2020 31(5): 107597 (IF: 7.8)
10. Balli M, Vitali F, Janiszewski A, Caluwé E, Cortés-Calabuig A, Carpentier S, Duelen R, Ronzoni F, Marcelis L, Bosisio FM, Bellazzi R, Luttun A, De Angelis MGC, Ceccarelli G, Lluis F, Sampaolesi M. Autologous micrograft accelerates endogenous wound healing response through ERK-induced cell migration. Cell Death Differ. 2020 27(5): 1520-1538 (IF: 15.828)
11. Rotini A, Martínez-Sarrà E, Duelen R, Costamagna D, Di Filippo ES, Giacomazzi G, Grosemans H, Fulle S, and Sampaolesi M. Aging affects the in vivo regenerative potential of human mesoangioblasts. Aging Cell. 2018 17(2) (IF: 7.346)
12. Giacomazzi G, Holvoet B, Trenson S, Caluwé E, Kravic B, Grosemans H, Cortés-Calabuig Á, Deroose CM, Huylebroeck D, Hashemolhosseini S, Janssens S, McNally E, Quattrocelli M, Sampaolesi M. MicroRNAs promote skeletal muscle differentiation of mesodermal iPSC-derived progenitors. Nat Commun. 2017; 8(1): 1249 (IF: 12.353)
13. Quattrocelli M, Swinnen M, Giacomazzi G, Camps J, Barthélemy I, Ceccarelli G, Caluwé E, Grosemans H, Thorrez L, Pelizzo G, Muijtjens M, Verfaillie CM, Blot S, Janssens S, Sampaolesi M. Mesodermal iPSC-derived progenitor cells functionally regenerate cardiac and skeletal muscle. J Clin Invest. 2015, 125(12): 4463-82 (IF: 12.6)
14. Sampaolesi M, Blot S, D'Antona G, Granger N, Tonlorenzi R, Innocenzi A, Mognol P, Thibaud JL, Galvez BG, Barthélémy I, Perani L, Mantero S, Guttinger M, Pansarasa O, Rinaldi C, Cusella De Angelis MG, Torrente Y, Bordignon C, Bottinelli R, Cossu G. Mesoangioblast stem cells ameliorate muscle function in dystrophic dogs. Nature 2006 444(7119): 574-9 (IF: 26.7)
15. Sampaolesi M, Torrente Y, Innocenzi A, Tonlorenzi R, D'Antona G, Pellegrino MA, Barresi R, Bresolin N, De Angelis MG, Campbell KP, Bottinelli R, Cossu G. Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science 2003 301(5632): 487-92 (IF: 29.2)

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