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33 results on '"Olgasi, Cristina"'

2. Factor VIII promotes angiogenesis and vessel stability regulating extracellular matrix proteins

Author

Olgasi, Cristina, primary, Cucci, Alessia, additional, Molineris, Ivan, additional, Assanelli, Simone, additional, Anselmi, Francesca, additional, Borsotti, Chiara, additional, Sgromo, Chiara, additional, Lauria, Andrea, additional, Merlin, Simone, additional, Walker, Gillian E., additional, Oliviero, Salvatore, additional, and Follenzi, Antonia, additional

Published
2024
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3. Current status and new avenues of stem cell-based preclinical and therapeutic approaches in amyotrophic lateral sclerosis

Author

Mazzini, Letizia, De Marchi, Fabiola, Buzanska, Leonora, Follenzi, Antonia, Glover, Joel Clinton, Gelati, Maurizio, Lombardi, Ivan, Maioli, Margherita, Mesa-Herrera, Fatima, Mitrečić, Dinko, Olgasi, Cristina, Pivoriūnas, Augustas, Sanchez-Pernaute, Rosario, Sgromo, Chiara, Zychowicz, Marzena, Vescovi, Angelo, and Ferrari, Daniela

Abstract

ABSTRACTIntroductionCell therapy development represents a critical challenge in amyotrophic lateral sclerosis (ALS) research. Despite more than 20 years of basic and clinical research, no definitive safety and efficacy results of cell-based therapies for ALS have been published.Areas coveredThis review summarizes advances using stem cells (SCs) in pre-clinical studies to promote clinical translation and in clinical trials to treat ALS. New technologies have been developed and new experimental in vitroand animal models are now available to facilitate pre-clinical research in this field and to determine the most promising approaches to pursue in patients. New clinical trial designs aimed at developing personalized SC-based treatment with biological endpoints are being defined.Expert opinionKnowledge of the basic biology of ALS and on the use of SCs to study and potentially treat ALS continues to grow. However, a consensus has yet to emerge on how best to translate these results into therapeutic applications. The selection and follow-up of patients should be based on clinical, biological, and molecular criteria. Planning of SC-based clinical trials should be coordinated with patient profiling genetically and molecularly to achieve personalized treatment. Much work within basic and clinical research is still needed to successfully transition SC therapy in ALS.

Published
2024
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6. Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A

Author

Esposito, Federica, Lyubenova, Hristiana, Tornabene, Patrizia, Auricchio, Stefano, Iuliano, Antonella, Nusco, Edoardo, Merlin, Simone, Olgasi, Cristina, Manni, Giorgia, Gargaro, Marco, Fallarino, Francesca, Follenzi, Antonia, Auricchio, Alberto, Esposito, Federica, Lyubenova, Hristiana, Tornabene, Patrizia, Auricchio, Stefano, Iuliano, Antonella, Nusco, Edoardo, Merlin, Simone, Olgasi, Cristina, Manni, Giorgia, Gargaro, Marco, Fallarino, Francesca, Follenzi, Antonia, and Auricchio, Alberto

Subjects
Genetic Vectors, haemophilia A, Genetic Therapy, Dependovirus, Hemophilia A, AAV vectors, Inteins, Trans-Splicing, AAV vector, Mice, Liver, Molecular Medicine, Animals, liver gene therapy, protein trans-splicing
Abstract

Liver gene therapy with adeno-associated viral (AAV) vectors is under clinical investigation for haemophilia A (HemA), the most common inherited X-linked bleeding disorder. Major limitations are the large size of the F8 transgene, which makes packaging in a single AAV vector a challenge, as well as the development of circulating anti-F8 antibodies which neutralise F8 activity. Taking advantage of split-intein-mediated protein trans-splicing, we divided the coding sequence of the large and highly secreted F8-N6 variant in two separate AAV-intein vectors whose co-administration to HemA mice results in the expression of therapeutic levels of F8 over time. This occurred without eliciting circulating anti-F8 antibodies unlike animals treated with the single oversized AAV-F8 vector under clinical development. Therefore, liver gene therapy with AAV-F8-N6 intein should be considered as a potential therapeutic strategy for HemA.

Published
2022

8. Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device

Author

Olgasi, Cristina, primary, Borsotti, Chiara, additional, Merlin, Simone, additional, Bergmann, Thorsten, additional, Bittorf, Patrick, additional, Adewoye, Adeolu Badi, additional, Wragg, Nicholas, additional, Patterson, Kelcey, additional, Calabria, Andrea, additional, Benedicenti, Fabrizio, additional, Cucci, Alessia, additional, Borchiellini, Alessandra, additional, Pollio, Berardino, additional, Montini, Eugenio, additional, Mazzuca, Delfina M., additional, Zierau, Martin, additional, Stolzing, Alexandra, additional, Toleikis, Philip.M., additional, Braspenning, Joris, additional, and Follenzi, Antonia, additional

Published
2021
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9. P.168: Biofabrication of a Functional Vascularized Endocrine Pancreas (VEP) for Type 1 Diabetes

Author

Citro, Antonio, primary, Neroni, Alessia, additional, Pignatelli, Cataldo, additional, Policardi, Martina, additional, Campo, Francesco, additional, Pellegrini, Silvia, additional, Dugnani, Erica, additional, Manenti, Fabio, additional, Valla, Libera, additional, Kemter, Elisabeth, additional, Marzinotto, Ilaria, additional, Olgasi, Cristina, additional, Follenzi, Antonia, additional, Lampasona, Vito, additional, Wolf, Eckhard, additional, and Piemonti, Lorenzo, additional

Published
2021
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12. Increase of palmitic acid concentration impairs endothelial progenitor cell and bone marrow--derived progenitor cell bioavailability: role of the STAT5/ PPARγ/transcriptional complex

Author

Trombetta, Antonella, Togliatto, Gabriele, Rosso, Arturo, Dentelli, Patrizia, Olgasi, Cristina, Cotogni, Paolo, and Brizzi, Maria Felice

Subjects
Fatty acids -- Physiological aspects -- Genetic aspects -- Research, Cell receptors -- Physiological aspects -- Genetic aspects -- Research, Type 2 diabetes -- Genetic aspects -- Diagnosis -- Care and treatment -- Research, Health
Abstract

Metabolic profiling of plasma nonesterified fatty acids discovered that palmitic acid (PA), a natural peroxisome proliferator-activated receptor γ (PPARγ) ligand, is a reliable type 2 diabetes biomarker. We investigated whether and how PA diabetic (d-PA) concentrations affected endothelial progenitor cell (EPC) and bone marrow-derived hematopoietic cell (BM-HC) biology. PA physiologic (n-PA) and d-PA concentrations were used. Proliferating cell nuclear antigen content and signal transducer and activator of transcription 5 (STAT5), PPARγ, cyclin D1, and [p21.sup.Waf] expression were evaluated. Small interfering RNA technology, gene reporter luciferase assay, electrophoretic mobility shift assay, chromatin immunoprecipitation assay, and coimmunoprecipitation were exploited. In vivo studies and migration assays were also performed, d-PA, unlike n-PA or physiological and diabetic oleic and stearic acid concentrations, impaired EPC migration and EPC/BM-HC proliferation through a PPARγ-mediated STAT5 transcription inhibition. This event did not prevent the formation of a STAT5/PPARγ transcriptional complex but was crucial for gene targeting, as [p21.sup.Waf] gene promoter, unlike cyclin D1, was the STAT5/PPARγ transcriptional target. Similar molecular events could be detected in EPCs isolated from type 2 diabetic patients. By expressing a constitutively activated STAT5 form, we demonstrated that STAT5 content is crucial for gene targeting and EPC fate. Finally, we also provide in vivo data that d-PA-mediated EPC dysfunction could be rescued by PPARγ blockade. These data provide first insights on how mechanistically d-PA drives EPC/BM-HC dysfunction in diabetes., Metabolomic, a recent tool exploited to explore the complex interactions of metabolites in livelihood systems, discloses the metabolic response to pathophysiologic stimuli or genetic alterations (1). In recent years, metabolomic [...]

Published
2013
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15. Patient-Specific iPSC-Derived Endothelial Cells Provide Long-Term Phenotypic Correction of Hemophilia A

Author

Olgasi, Cristina, primary, Talmon, Maria, additional, Merlin, Simone, additional, Cucci, Alessia, additional, Richaud-Patin, Yvonne, additional, Ranaldo, Gabriella, additional, Colangelo, Donato, additional, Di Scipio, Federica, additional, Berta, Giovanni N., additional, Borsotti, Chiara, additional, Valeri, Federica, additional, Faraldi, Francesco, additional, Prat, Maria, additional, Messina, Maria, additional, Schinco, Piercarla, additional, Lombardo, Angelo, additional, Raya, Angel, additional, and Follenzi, Antonia, additional

Published
2018
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16. A functional assay for the clinical annotation of genetic variants of uncertain significance in Diamond-Blackfan anemia

Author

Aspesi, Anna, primary, Betti, Marta, additional, Sculco, Marika, additional, Actis, Chiara, additional, Olgasi, Cristina, additional, Wlodarski, Marcin W., additional, Vlachos, Adrianna, additional, Lipton, Jeffrey M., additional, Ramenghi, Ugo, additional, Santoro, Claudio, additional, Follenzi, Antonia, additional, Ellis, Steven R., additional, and Dianzani, Irma, additional

Published
2018
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17. Identification and functional characterization of a novel splicing variant in the F8coagulation gene causing severe hemophilia A

Author

Famà, Rosella, Borroni, Ester, Zanolini, Diego, Merlin, Simone, Bruscaggin, Valentina, Walker, Gillian E., Olgasi, Cristina, Babu, Deepak, Agnelli Giacchello, Jacopo, Valeri, Federica, Giordano, Mara, Borchiellini, Alessandra, and Follenzi, Antonia

Abstract

We have identified a synonymous F8variation in a severe hemophilia A (HA) patient who developed inhibitors following factor VIII (FVIII) prophylaxis. The unreported c.6273 G > A variant targets the consensussplicing site of exon 21.

Published
2020
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23. Vaccination to kitL: a new approach to inhibit tumor angiogenesis

Author

Olgasi, Cristina, Dentelli, Patrizia, Trombetta, Antonella, Rosso, Arturo, Togliatto, Gabriele Maria, Iavello, Alessandra, Castelli, Ada, Gili, Maddalena, Alberto, Orsello, Simone, Prestigio, Forni, Guido, Cavallo, Federica, and Brizzi, Maria Felice

Published
2011

24. 179. Correcting the Bleeding Phenotype in Hemophilia Ausing Lentivirally FVIII-Corrected Endothelial Cells Differentiated from Hemophilic Induced Pluripotent Stem Cell (iPSC)

Author

Talmon, Maria, primary, Olgasi, Cristina, additional, Merlin, Simone, additional, Lombardo, Angelo, additional, Valeri, Federica, additional, Raya, Angel, additional, Messina, Maria, additional, Colangelo, Donato, additional, Schinco, Piercarla, additional, and Follenzi, Antonia, additional

Published
2015
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28. DNA vaccination against membrane-bound Kit ligand: A new approach to inhibiting tumour growth and angiogenesis

Author

Olgasi, Cristina, primary, Dentelli, Patrizia, additional, Rosso, Arturo, additional, Iavello, Alessandra, additional, Togliatto, Gabriele, additional, Toto, Valentina, additional, Liberatore, Marcella, additional, Barutello, Giuseppina, additional, Musiani, Piero, additional, Cavallo, Federica, additional, and Brizzi, Maria Felice, additional

Published
2014
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32. Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device.

Author

Olgasi C, Borsotti C, Merlin S, Bergmann T, Bittorf P, Adewoye AB, Wragg N, Patterson K, Calabria A, Benedicenti F, Cucci A, Borchiellini A, Pollio B, Montini E, Mazzuca DM, Zierau M, Stolzing A, Toleikis PM, Braspenning J, and Follenzi A

Abstract

Hemophilia A (HA) is a rare bleeding disorder caused by deficiency/dysfunction of the FVIII protein. As current therapies based on frequent FVIII infusions are not a definitive cure, long-term expression of FVIII in endothelial cells through lentiviral vector (LV)-mediated gene transfer holds the promise of a one-time treatment. Thus, here we sought to determine whether LV-corrected blood outgrowth endothelial cells (BOECs) implanted through a prevascularized medical device (Cell Pouch) would rescue the bleeding phenotype of HA mice. To this end, BOECs from HA patients and healthy donors were isolated, expanded, and transduced with an LV carrying FVIII driven by an endothelial-specific promoter employing GMP-like procedures. FVIII-corrected HA BOECs were either directly transplanted into the peritoneal cavity or injected into a Cell Pouch implanted subcutaneously in NSG-HA mice. In both cases, FVIII secretion was sufficient to improve the mouse bleeding phenotype. Indeed, FVIII-corrected HA BOECs reached a relatively short-term clinically relevant engraftment being detected up to 16 weeks after transplantation, and their genomic integration profile did not show enrichment for oncogenes, confirming the process safety. Overall, this is the first preclinical study showing the safety and feasibility of transplantation of GMP-like produced LV-corrected BOECs within an implantable device for the long-term treatment of HA., Competing Interests: K.P., D.M.M., and P.M.T. are/have been employees of Sernova Corp., which holds the patent US20190240375A1., (© 2021 The Authors.)

Published
2021
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33. Regulatory-Compliant Validation of a Highly Sensitive qPCR for Biodistribution Assessment of Hemophilia A Patient Cells.

Author

Bittorf P, Bergmann T, Merlin S, Olgasi C, Pullig O, Sanzenbacher R, Zierau M, Walles H, Follenzi A, and Braspenning J

Abstract

The investigation of the biodistribution profile of a cell-based medicinal product is a pivotal prerequisite to allow a factual benefit-risk assessment within the non-clinical to clinical translation in product development. Here, a qPCR-based method to determine the amount of human DNA in mouse DNA was validated according to the guidelines of the European Medicines Agency and the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. Furthermore, a preclinical worst-case scenario study was performed in which this method was applied to investigate the biodistribution of 2 × 10 6 intravenously administered, genetically modified, blood outgrowth endothelial cells from hemophilia A patients after 24 h and 7 days. The validation of the qPCR method demonstrated high accuracy, precision, and linearity for the concentration interval of 1:1 × 10 3 to 1:1 × 10 6 human to mouse DNA. The application of this method in the biodistribution study resulted in the detection of human genomes in four out of the eight investigated organs after 24 h. After 7 days, no human DNA was detected in the eight organs analyzed. This biodistribution study provides mandatory data on the toxicokinetic safety profile of an actual candidate cell-based medicinal product. The extensive evaluation of the required validation parameters confirms the applicability of the qPCR method for non-clinical biodistribution studies., (© 2020 The Authors.)

Published
2020
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