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Your search keyword '"Roncarolo, M"' showing total 15 results
Start Over Author "Roncarolo, M" Publisher springer science and business media llc
15 results on '"Roncarolo, M"'

2. Erratum: BAT2 and BAT3 polymorphisms as novel genetic risk factors for rejection after HLA-related SCT

Author

Piras, I S, primary, Angius, A, additional, Andreani, M, additional, Testi, M, additional, Lucarelli, G, additional, Floris, M, additional, Marktel, S, additional, Ciceri, F, additional, La Nasa, G, additional, Fleischhauer, K, additional, Roncarolo, M G, additional, Bulfone, A, additional, Gregori, S, additional, and Bacchetta, R, additional

Published
2014
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4. Sirolimus-based graft-versus-host disease prophylaxis promotes the in vivo expansion of regulatory T cells and permits peripheral blood stem cell transplantation from haploidentical donors

Author

Peccatori, J, primary, Forcina, A, additional, Clerici, D, additional, Crocchiolo, R, additional, Vago, L, additional, Stanghellini, M T L, additional, Noviello, M, additional, Messina, C, additional, Crotta, A, additional, Assanelli, A, additional, Marktel, S, additional, Olek, S, additional, Mastaglio, S, additional, Giglio, F, additional, Crucitti, L, additional, Lorusso, A, additional, Guggiari, E, additional, Lunghi, F, additional, Carrabba, M, additional, Tassara, M, additional, Battaglia, M, additional, Ferraro, A, additional, Carbone, M R, additional, Oliveira, G, additional, Roncarolo, M G, additional, Rossini, S, additional, Bernardi, M, additional, Corti, C, additional, Marcatti, M, additional, Patriarca, F, additional, Zecca, M, additional, Locatelli, F, additional, Bordignon, C, additional, Fleischhauer, K, additional, Bondanza, A, additional, Bonini, C, additional, and Ciceri, F, additional

Published
2014
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7. Second hematopoietic SCT in patients with thalassemia recurrence following rejection of the first graft

Author

Gaziev, J, primary, Sodani, P, additional, Lucarelli, G, additional, Polchi, P, additional, Marktel, S, additional, Paciaroni, K, additional, Marziali, M, additional, Isgrò, A, additional, Simone, M D, additional, Roveda, A, additional, Montuoro, A, additional, Lanti, A, additional, Alfieri, C, additional, De Angelis, G, additional, Gallucci, C, additional, Ciceri, F, additional, and Roncarolo, M G, additional

Published
2008
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8. Multiple BM harvests in pediatric donors for thalassemic siblings: safety, efficacy and ethical issues

Author

Biral, E, primary, Chiesa, R, additional, Cappelli, B, additional, Roccia, T, additional, Frugnoli, I, additional, Noè, A, additional, Soliman, C, additional, Fiori, R, additional, Cursi, L, additional, Cattaneo, F, additional, Evangelio, C, additional, Miniero, R, additional, Ciceri, F, additional, Roncarolo, M G, additional, and Marktel, S, additional

Published
2008
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12. The X-linked lymphoproliferative-disease gene product SAP regulates signals induced through the co-receptor SLAM

Author

Sayos, J., primary, Wu, C., additional, Morra, M., additional, Wang, N., additional, Zhang, X., additional, Allen, D., additional, van Schaik, S., additional, Notarangelo, L., additional, Geha, R., additional, Roncarolo, M. G., additional, Oettgen, H., additional, De Vries, J. E., additional, Aversa, G., additional, and Terhorst, C., additional

Published
1998
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13. Ligand for FLT3/FLK2 receptor tyrosine kinase regulates growth of haematopoietic stem cells and is encoded by variant RNAs

Author

Hannum, C., primary, Culpepper, J., additional, Campbell, D., additional, McClanahan, T., additional, Zurawski, S., additional, Kastelein, R., additional, Bazan, J. F., additional, Hudak, S., additional, Wagner, J., additional, Mattson, J., additional, Luh, J., additional, Duda, G., additional, Martina, N., additional, Peterson, D, additional, Menon, S., additional, Shanafelt, A., additional, Muench, M., additional, Kelner, G., additional, Namikawa, R., additional, Rennick, D., additional, Roncarolo, M.-G., additional, Zlotnik, A., additional, Rosnet, O., additional, Dubreuil, P., additional, Birnbaum, D., additional, and Lee, F., additional

Published
1994
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14. Gene correction for SCID-X1 in long-term hematopoietic stem cells

Author

Beruh Dejene, Sruthi Mantri, Mara Pavel-Dinu, Harry L. Malech, Ciaran M. Lee, Niraj Punjya, Matthew A. Inlay, Volker Wiebking, Camille Sindhu, Waracharee Srifa, Carmencita E. Nicolas, Kenneth I. Weinberg, Maria Grazia Roncarolo, Gang Bao, Nivedita Saxena, Suk See DeRavin, Eric J. Kildebeck, Matthew H. Porteus, Pavel-Dinu, M., Wiebking, V., Dejene, B. T., Srifa, W., Mantri, S., Nicolas, C. E., Lee, C., Bao, G., Kildebeck, E. J., Punjya, N., Sindhu, C., Inlay, M. A., Saxena, N., Deravin, S. S., Malech, H., Roncarolo, M. G., Weinberg, K. I., and Porteus, M. H.

Subjects
Genome instability, Male, 0301 basic medicine, Time Factors, medicine.medical_treatment, CD34, Codon, Initiator, General Physics and Astronomy, Antigens, CD34, 02 engineering and technology, Hematopoietic stem cell transplantation, X-Linked Combined Immunodeficiency Diseases, Genome, Mice, 0302 clinical medicine, Genome editing, Parvovirinae, Transduction, Genetic, lcsh:Science, Gene Editing, 0303 health sciences, education.field_of_study, Multidisciplinary, Hematopoietic Stem Cell Transplantation, Exons, Fetal Blood, 021001 nanoscience & nanotechnology, Healthy Volunteers, 3. Good health, Haematopoiesis, 030220 oncology & carcinogenesis, Stem cell, 0210 nano-technology, Interleukin Receptor Common gamma Subunit, DNA, Complementary, Science, Genetic Vectors, Primary Cell Culture, Transplantation, Heterologous, Population, Biology, Article, General Biochemistry, Genetics and Molecular Biology, Cell Line, 03 medical and health sciences, medicine, Animals, Humans, education, Gene, 030304 developmental biology, Severe combined immunodeficiency, Transplantation Chimera, General Chemistry, medicine.disease, Hematopoietic Stem Cells, Transplantation, 030104 developmental biology, Mutation, Cancer research, lcsh:Q, CRISPR-Cas Systems
Abstract

Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. Here we describe an approach for X-linked sSevere cCombined iImmunodeficiency (SCID-X1) using targeted integration of a cDNA into the endogenous start codon to functionally correct disease-causing mutations throughout the gene. Using a CRISPR-Cas9/AAV6 based strategy, we achieve up to 20% targeted integration frequencies in LT-HSCs. As measures of the lack of toxicity we observe no evidence of abnormal hematopoiesis following transplantation and no evidence of off-target mutations using a high-fidelity Cas9 as a ribonucleoprotein complex. We achieve high levels of targeting frequencies (median 45%) in CD34+ HSPCs from six SCID-X1 patients and demonstrate rescue of lymphopoietic defect in a patient derived HSPC population in vitro and in vivo. In sum, our study provides specificity, toxicity and efficacy data supportive of clinical development of genome editing to treat SCID-Xl., Gene correction in hematopoietic stem cells could be a powerful way to treat monogenic diseases of the blood and immune system. Here the authors develop a strategy using CRISPR-Cas9 and an aAdeno-Associated vVirus(AAV)-delivered IL2RG cDNA to correct X-linked sSevere Ccombined iImmunodeficiency (SCID-X1) with a high success rate.

Published
2019
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15. Coexpression of CD49b and LAG-3 identifies human and mouse T regulatory type 1 cells

Author

Clelia Di Serio, Mauro Pala, Rosa Bacchetta, Silvia Gregori, Leonie Brockmann, Nicola Gagliani, Richard A. Flavell, Filippo Trentini, Samuel Huber, Paula Licona-Limón, Chiara F. Magnani, Marco Andreani, Monica E. Gianolini, Maria Grazia Roncarolo, Binggege Guo, Alessandro Bulfone, De'Broski R. Herbert, Gagliani, N, Magnani, C, Huber, S, Gianolini, M, Pala, M, Licona Limon, P, Guo, B, Herbert, D, Bulfone, A, Trentini, F, Di Serio, C, Bacchetta, R, Andreani, M, Brockmann, L, Gregori, S, Flavell, R, Roncarolo, M, Magnani, Cf, Gianolini, Me, Herbert, Dr, DI SERIO, Mariaclelia, Flavell, Ra, and Roncarolo, MARIA GRAZIA

Subjects
gene expression, t cells, non parametric tests, medicine.medical_treatment, Integrin alpha2, Cell Separation, Hematopoietic stem cell transplantation, Biology, T-Lymphocytes, Regulatory, General Biochemistry, Genetics and Molecular Biology, CD49b, Th17 Cell, Transcriptome, Cell therapy, Mice, Antigen, Antigens, CD, Nippostrongylu, medicine, Animals, Humans, Gene, Strongylida Infections, Biochemistry, Genetics and Molecular Biology (all), Animal, Strongylida Infection, Medicine (all), General Medicine, Lymphocyte Activation Gene 3 Protein, Molecular biology, In vitro, Cell biology, Mice, Inbred C57BL, Gene expression profiling, Th17 Cells, Nippostrongylus, Human
Abstract

CD4+ type 1 T regulatory (Tr1) cells are induced in the periphery and have a pivotal role in promoting and maintaining tolerance. The absence of surface markers that uniquely identify Tr1 cells has limited their study and clinical applications. By gene expression profiling of human Tr1 cell clones, we identified the surface markers CD49b and lymphocyte activation gene 3 (LAG-3) as being stably and selectively coexpressed on mouse and human Tr1 cells. We showed the specificity of these markers in mouse models of intestinal inflammation and helminth infection and in the peripheral blood of healthy volunteers. The coexpression of CD49b and LAG-3 enables the isolation of highly suppressive human Tr1 cells from in vitro anergized cultures and allows the tracking of Tr1 cells in the peripheral blood of subjects who developed tolerance after allogeneic hematopoietic stem cell transplantation. The use of these markers makes it feasible to track Tr1 cells in vivo and purify Tr1 cells for cell therapy to induce or restore tolerance in subjects with immune-mediated diseases. © 2013 Nature America, Inc. All rights reserved

Published
2013
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