The Molecular and Regenerative Medicine Laboratory



Building 8, Laboratory 1.19

Department of Biomedical Sciences and Medicine.

University of Algarve

Phone: +351 289 800 094

Team Members

Gustavo Tiscornia (Principal Investigator)
Marta Vitorino (Postdoc Student)
Dino Matias (PhD student)
Diogo Prata (MSs Student)
Goncalo Pinheiro (Research Assistant)

About the MRML

At the Molecular and Regenerative Medicine Laboratory (MRML), we are interested in studying mechanisms of pathogenesis and pharmacological compound testing for Gaucher’s Disease. We have developed a number of induced pluripotent stem cell lines derived from GD patient fibroblasts with different genotypes. We are able to differentiate them efficiently to neuronal phenotypes. Using this system, we have created a cell culture platform for the evaluation of chemical chaperones, and are currently evaluating a number of promising candidates.

A second line of research focuses on mammalian regeneration. We have developed an ear wound model in African spiny mice (Acomys). Acomys close ear wounds in 8 weeks by regenerating dermis, epidermis, cartilage, hair follicles and adipose tissue in the original tissue architecture. We are studying the molecular and cellular mechanisms underlying regeneration in this species.

Publications (2000-2017)

2017:  Induced Pluripotent Stem Cell Modeling of Gaucher’s Disease: What Have We Learned? D. Matias and G. Tiscornia. Int J Mol Sci Apr; 18 (4): 888.

2017:  Coenzyme Q10 partially restores pathological alterations in a macrophage model of Gaucher disease by M de la Mata , D. Cotán, M. Oropesa-Ávila, M. Villanueva-Paz, I. de Lavera, M. Álvarez-Córdoba , R. Luzón-Hidalgo, J.M. Suárez-Rivero, G. Tiscornia, and J.A. Sánchez-Alcázar. Orphanet J Rare Dis. Feb 6;12 (1):23.

2016: Mitochondrial Dysfunction in Lysosomal Storage Disorders Diseases by M. de la Mata, D. Cotán, M. Villanueva-Paz, I. de Lavera, M. Álvarez-Córdoba, R. Luzón-Hidalgo, J.M. Suárez-Rivero, G. Tiscornia and M. Oropesa-Ávila. Diseases 4 (4), 31.

2016:  Ear Regeneration in the African Spiny Mouse (Acomys cahirinus), by D. Matias, AR Martins, I. Casanellas Mercado, A. Brito Ova, D. Powers and G. Tiscornia. Regeneration  3 (1): 52- 61.

2015:  Highly pH-Responsive Pharmacological Chaperones for Mutant Glycosidase Enhancement, by T. Mena-Barragán, A. Narita, D. Matias, G. Tiscornia, E. Nanba, K. Ohno, Y. Suzuki, K. Higaki, J.M. García Fernández and C Ortiz Mellet. Angewandte Chemie, Int. Ed., 54, 1 – 6.

2013:  MicroRNA regulation of Stem Cell fate and Reprogramming, by E. Lorenzo Vivas, G. Tiscornia and J.C. Ispizua Belmonte, Chapter 2 in ‘MicroRNAs in Medicine’, p 27-40, ISBN978-1-118-30039-8, John Wiley and Sons, Inc., Hoboken, New Jersey.

2013:  G. Tiscornia, E. Lorenzo Vivas, L. Matalonga,  I. Berniakovich, M. Barragan Monasterio, C. Eguizabal, L. Gort, F. Gonzalez, C. Ortiz Mellet, J.M.  García Fernández, A. Ribes, A. Veiga and J.C. Izpisúa Belmonte. Neuronopathic Gaucher’s disease: induced pluripotent stem cells for disease modelling and testing chaperone activity of small compounds. Human Molecular Genetics 22(4): 633-45.

2011: G. Tiscornia, E. Lorenzo Vivas and J. C. Izpisúa Belmonte. Diseases in a Dish: modeling human disease with induced pluripotent stem cells. Nature Medicine Vol.17(12): 1570-1576.

2011: G. Tiscornia, N. Monserrat and J.C. Izpisúa Belmonte. Modelling Long QT Syndrome in iPS cells: Be still, my beating heart…, Circ. Res. 108: 648-649.

2010: G. Tiscornia and J.C. Izpisúa Belmonte. MicroRNAs in Stem Cell Function and Fate, Genes & Dev. 24: 2732-2741.

2009: Generation of disease-free Hematopoietic Progenitors from Fanconi anemia-specific Induced Pluripotent Stem Cells, by A. Raya, I. Rodríguez-Pizà, G. Guenechea, R. Vassena, S.Navarro, M.J. Barrero, A. Consiglio, P. Río, E. Sleep, G. Tiscornia, E. Garreta, T. Aasen, A. Veiga, I. M. Verma, J. Bueren and J. C. Izpisúa Belmonte, Nature 460, 53-59.

2009: Generation of Mouse Induced Pluripotent Stem Cells by Transient Expression of a Single Non-Viral Polycistronic Vector, by F. González, M. Barragán Monasterio, G. Tiscornia, N. Montserrat Pulido, R. Vassena, L. Battle Morera, I. Rodriguez Pizá and  J.C. Izpisúa Belmonte, PNAS 106:8918-8922.

2008:  Efficient and rapid generation of induced pluripotent stem cells from human keratinocytes, by T. Aasen, A. Raya, M.J. Barrero, E. Garreta, A. Consiglio, F. Gonzalez, R. Vassena, J. Bilić, V. Pekarik, G. Tiscornia, M. Edel, S. Boué & J. C. Izpisúa Belmonte, Nature Biotechnology 26, p1276-1284.

2006: Design and cloning of lentiviral vectors expressing small interfering siRNAs, by G. Tiscornia, O. Singer and I. Verma, Nature Protocols vol1 no1, p 234-240.

2006:  Production and purification of lentiviral vectors, by G. Tiscornia, O. Singer and I. Verma, Nature Protocols vol1 no1, p 241-245.

2006:  Rapid generation of Knockdown Transgenic Mice by Silencing Lentiviral Vectors, by O. Singer, G. Tiscornia, M. Ikawa and I Verma, Nature Protocols vol1 no1, p286-292.

2005:  Development of Lentiviral Vectors Expressing siRNA, by G. Tiscornia, O. Singer and I. Verma. Chapter 3 in ‘Gene Transfer: Delivery and Expression of DNA and RNA’, p23-34. ISBN: 13:978-0-87969-765. Cold Spring Harbor Laboratory Press.

2005: Knockdown transgenic mice generated by Silencing Lentiviral Vectors, by O. Singer, G. Tiscornia and I. Verma. Chapter 75 in ‘Gene Transfer: Delivery and Expression of DNA and RNA’, p755-759. ISBN: 13:978-0-87969-765. Cold Spring Harbor Laboratory Press.

2004:  RNAi: siRNA delivery by Lentiviral Vectors: Design and Applications. Singer, G. Tiscornia and I. Verma. Chapter 12 in ‘RNA Interference Technology’ p 174-185. ISBN: 0-521-83677-8, Cambridge University Press.

2004:  CRE recombinase Inducible RNA Interference Mediated by Lentiviral Vectors, by G. Tiscornia, V. Tergaonkar, F. Galimi and Inder Verma. Proc. Natl. Acad. Sci. USA, 2004, vol. 101 (19): 7347- 7351.

2003:  A general method for Gene Knockdown in Mice using Lentiviral Vectors expressing small interfering RNA, by G. Tiscornia, O. Singer, M. Ikawa and  I. Verma. Proc. Natl. Acad. Sci. USA, 2003, vol. 100 (4):1844-1848.

2001:  Purification and Characterization of Human Cell-Cell Adhesion Molecule 1 (C-CAM1) Expressed in Insect Cells, by D. Phan, E. Han, G. Birrell, S. Bonnal, L. Duggan, Esumi N, H. Gutstein, R. Li. S. Lopato, A. Manogaran, E.S. Pollak, A. Ray, P.P. Reddi, P. Struffi, G. Tiscornia, L.A. Ximenez-Fyvie, H. Zhang y S.H. Lin. Protein Expression and Purification 2001, vol. 21, p343-351.

2000:  Myotonic Dystrophy: the Role of the CUG Triplet Repeats in Splicing of a Novel DMPK Exon and Altered Cytoplasmic DMPK mRNA Isoform Ratios, by G. Tiscornia and M. Mahadevan. Molecular Cell Vol 5, p959-967.




Representative Images

Project Grants

2014: FCT Portugal (PTDC/BIA-ANM/0697/2014): Regeneration in the African Spiny Mouse.

2012: Genzyme Young Investigator Award, Development of a Gaucher’s Disease iPSc model.

2012: Marie Curie Career Integration Grant, FP7-PEOPLE-2011-CIG (# FP7-304225).

2007: Ramón y Cajal Grant, Ministerio Ciencia e Innovacion de España, The Role of microRNAs in Embryonic Stem Cells.

2004: Research Grant: Lentiviral Delivery of siRNA against huntingtin, Hereditary Disease Foundation, New York, USA.

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