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Resultados globales: 5 registros encontrados en 0.02 segundos.
Artículos, Encontrados 4 registros
Documentos de investigación, Encontrados 1 registros
Artículos Encontrados 4 registros  
1.
18 p, 30.1 MB Preclinical Development of a Therapy for Chronic Traumatic Spinal Cord Injury in Rats Using Human Wharton's Jelly Mesenchymal Stromal Cells : Proof of Concept and Regulatory Compliance / Vives Armengol, Joaquim (Universitat Autònoma de Barcelona. Departament de Medicina) ; Hernández, Joaquim (Universitat Autònoma de Barcelona. Departament de Biologia Cel·lular, de Fisiologia i d'Immunologia) ; Mirabel, Clémentine (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Puigdomenech-poch, Maria (Universitat Autònoma de Barcelona. Departament de Biologia Cel·lular, de Fisiologia i d'Immunologia) ; Romeo-Guitart, David (Universitat Autònoma de Barcelona. Departament de Biologia Cel·lular, de Fisiologia i d'Immunologia) ; Marmolejo Martínez-Artesero, Sara (Universitat Autònoma de Barcelona. Departament de Biologia Cel·lular, de Fisiologia i d'Immunologia) ; Cabrera-Pérez, Raquel (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Jaramillo, Jessica (Universitat Autònoma de Barcelona. Departament de Biologia Cel·lular, de Fisiologia i d'Immunologia) ; Kumru, Hatice (Universitat Autònoma de Barcelona. Departament de Medicina) ; García-lópez, Joan (Universitat Autònoma de Barcelona. Departament de Medicina) ; Vidal-Samsó, Joan (Universitat Autònoma de Barcelona. Departament de Medicina) ; Navarro, X. (Xavier) (Universitat Autònoma de Barcelona. Departament de Biologia Cel·lular, de Fisiologia i d'Immunologia) ; Coll-Bonet, Ruth (Banc de Sang i Teixits)
(1) Background: the use of Mesenchymal Stromal Cells (MSC) in emerging therapies for spinal cord injury (SCI) hold the potential to improve functional recovery. However, the development of cell-based medicines is challenging and preclinical studies addressing quality, safety and efficacy must be conducted prior to clinical testing; (2) Methods: herein we present (i) the characterization of the quality attributes of MSC from the Wharton's jelly (WJ) of the umbilical cord, (ii) safety of intrathecal infusion in a 3-month subchronic toxicity assessment study, and (iii) efficacy in a rat SCI model by controlled impaction (100 kdynes) after single (day 7 post-injury) and repeated dose of 1 × 10 MSC,WJ (days 7 and 14 post-injury) with 70-day monitoring by electrophysiological testing, motor function assessment and histology evaluation; (3) Results: no toxicity associated to MSC,WJ infusion was observed. [...]
2022 - 10.3390/cells11142153
Cells, Vol. 11 Núm. 14 (7-2 2022) , p. 2153  
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2.
12 p, 2.8 MB Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides / Vila-Julià, Ferran (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Cabrera-Pérez, Raquel (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Cámara, Yolanda (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Molina-Berenguer, Miguel (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Lope-Piedrafita, Silvia (Universitat Autònoma de Barcelona. Servei de Ressonància Magnètica Nuclear) ; Hirano, Michio (Department of Neurology, H. Houston Merritt Neuromuscular Research Center, Columbia University Irving Medical Center) ; Mingozzi, Federico (Spark Therapeutics) ; Torres-Torronteras, Javier (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Martí, Ramon A. (Hospital Universitari Vall d'Hebron. Institut de Recerca)
Preclinical studies have shown that gene therapy is a feasible approach to treat mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). However, the genetic murine model of the disease (Tymp/Upp1 double knockout, dKO) has a limited functional phenotype beyond the metabolic imbalances, and so the studies showing efficacy of gene therapy have relied almost exclusively on demonstrating correction of the biochemical phenotype. [...]
2020 - 10.1016/j.ebiom.2020.103133
EBioMedicine, Vol. 62 (november 2020)  
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3.
11 p, 7.8 MB Osteogenic commitment of Wharton's jelly mesenchymal stromal cells : Mechanisms and implications for bioprocess development and clinical application / Cabrera-Pérez, Raquel (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Monguió-Tortajada, Marta (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ; Gámez-Valero, Ana (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ; Rojas-Márquez, R. (Centre de Regulació Genòmica) ; Borràs i Serres, Francesc Enric (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ; Rudilla, F. (Institut Germans Trias i Pujol. Hospital Universitari Germans Trias i Pujol) ; Vives Armengol, Joaquim (Universitat Autònoma de Barcelona. Departament de Medicina)
Background: Orthopaedic diseases are one of the major targets for regenerative medicine. In this context, Wharton's jelly (WJ) is an alternative source to bone marrow (BM) for allogeneic transplantation since its isolation does not require an invasive procedure for cell collection and does not raise major ethical concerns. [...]
2019 - 10.1186/s13287-019-1450-3
Stem cell research & therapy, Vol. 10 Núm. 1 (28 2019) , p. 356  
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4.
14 p, 2.3 MB Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of MNGIE / Yadak, Rana (Department of Hematology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Cabrera-Pérez, Raquel (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Torres-Torronteras, Javier (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Bugiani, Marianna (Department of Pathology, VU University Medical Center, Amsterdam, the Netherlands) ; Haeck, Joost C. (Department of Radiology & Nuclear Medicine, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Huston, Marshall W. (Department of Neurology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Bogaerts, Elly (Department of Clinical Genetics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Goffart, Steffi (Department of Biology, University of Eastern Finland, Joensuu, Finland) ; Jacobs, Edwin H. (Department of Clinical Genetics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Stok, Merel (Department of Pediatrics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Leonardelli, Lorena (San Raffaele Telethon Institute for Gene Therapy (Milà, Itàlia)) ; Biasco, Luca (University College of London (UCL), Great Ormond Street Institute of Child Health (ICH), London, UK) ; Verdijk, Robert M. (Department of Pathology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Bernsen, Monique R. (Department of Radiology & Nuclear Medicine, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Ruijter, George (Department of Clinical Genetics, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Martí, Ramon A. (Hospital Universitari Vall d'Hebron. Institut de Recerca) ; Wagemaker, Gerard (Raisa Gorbacheva Memorial Research Institute for Pediatric Oncology and Hematology, Saint Petersburg, Russia) ; van Til, Niek P. (Laboratory of Translational Immunology, University Medical Center Utrecht, Utrecht, the Netherlands) ; de Coo, Irenaeus F. M. (Department of Neurology, Erasmus University Medical Center, Rotterdam, the Netherlands) ; Universitat Autònoma de Barcelona
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by thymidine phosphorylase (TP) deficiency resulting in systemic accumulation of thymidine (d-Thd) and deoxyuridine (d-Urd) and characterized by early-onset neurological and gastrointestinal symptoms. [...]
2018 - 10.1016/j.omtm.2018.01.001
Molecular Therapy. Methods & Clinical Development, Vol. 8 (january 2018) , p. 152-165  
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Documentos de investigación Encontrados 1 registros  
1.
239 p, 7.3 MB Terapia génica para el MNGIE : estudio comparativo de diferentes vectores adeno-asociados en el modelo preclínico de la enfermedad / Cabrera-Pérez, Raquel ; Martí, Ramon A., dir. ; Vila Bover, Miquel, dir. (Universitat Autònoma de Barcelona. Departament de Bioquímica i de Biologia Molecular) ; Universitat Autònoma de Barcelona. Departament de Bioquímica i de Biologia Molecular
El MNGIE (encefalomiopatía neurogastrointestinal mitocondrial) es una enfermedad rara de herencia autosómica recesiva que provoca afectación de la función muscular, neuronal y gastrointestinal y cuya esperanza de vida se sitúa en torno a los 37 años. [...]
MNGIE (Mitochondrial NeuroGastroIntestinal Encephalomyopathy) is a rare autosomal recessive disease characterized by muscular, neuronal and gastrointestinal symptoms. The average life-span of MNGIE patients is 37 years. [...]
[Barcelona] : Universitat Autònoma de Barcelona, 2017.  
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