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1Academic Journal
المؤلفون: Magnani, A., Semeraro, M., Adam, F., Booth, C., Dupré, Loïc, Morris, E., Gabrion, A., Roudaut, C., Borgel, D., Toubert, A., Clave, E., Abdo, C., Gorochov, G., Petermann, R., Guiot, M., Miyara, M., Moshous, D., Magrin, E., Denis, A., Suarez, F., Lagresle, C., Roche, A., Everett, J., Trinquand, A., Guisset, M., Bayford, J. Xu, Hacein-Bey-Abina, S., Kauskot, A., Elfeky, R., Rivat, C., Abbas, S., Gaspar, H., Macintyre, E., Picard, C., Bushman, F., Galy, A., Fischer, A., Six, E., Thrasher, A., Cavazzana, M.
المساهمون: Département de Biothérapie CHU Necker, Hôpital Necker - Enfants Malades AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Centre d'investigation clinique Biothérapie CHU Pitié-Salpêtrière (CIC-BTi), Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), CIC - Mère Enfant Necker Cochin Paris Centre (CIC 1419), Hôpital Cochin AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité), Hémostase, Inflammation, Thrombose (HITH - U1176 Inserm - CHU Bicêtre), Institut National de la Santé et de la Recherche Médicale (INSERM)-Hôpital Bicêtre AP-HP, Le Kremlin-Bicêtre, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Université Paris-Saclay, Great Ormond Street Hospital for Children London (GOSH), Great Ormond Street Institute of Child Health (UCL), University College of London London (UCL), Institut Toulousain des Maladies Infectieuses et Inflammatoires (Infinity), Université Toulouse III - Paul Sabatier (UT3), Université de Toulouse (UT)-Université de Toulouse (UT)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases (LBI-RUD), Medizinische Universität Wien = Medical University of Vienna, NHS Foundation Trust London, The Royal Marsden, Généthon, Service d'immuno-hématologie pédiatrique CHU Necker, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Necker - Enfants Malades AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Hopital Saint-Louis AP-HP (AP-HP), Ecotaxie, microenvironnement et développement lymphocytaire (EMily (UMR_S_1160 / U1160)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité), Institut Necker Enfants-Malades (INEM - UM 111 (UMR 8253 / U1151)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité), CHU Pitié-Salpêtrière AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Centre d'Immunologie et des Maladies Infectieuses (CIMI), Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Institut National de la Transfusion Sanguine Paris (INTS), Imagine - Institut des maladies génétiques (IHU) (Imagine - U1163), University of Pennsylvania School of Veterinary Medicine, Unité de Technologies Chimiques et Biologiques pour la Santé (UTCBS - UM 4 (UMR 8258 / U1267)), Institut National de la Santé et de la Recherche Médicale (INSERM)-Institut de Chimie - CNRS Chimie (INC-CNRS)-Centre National de la Recherche Scientifique (CNRS)-Université Paris Cité (UPCité), Hôpital Bicêtre AP-HP, Le Kremlin-Bicêtre, Collège de France (CdF (institution)), ANR-10-IAHU-0001,Imagine,Institut Hospitalo-Universitaire Imagine(2010), European Project: 693762,GENEFORCURE
المصدر: ISSN: 1078-8956.
مصطلحات موضوعية: MESH: Clinical Trials, Phase I as Topic, Phase II as Topic, MESH: Genetic Therapy / methods, MESH: Genetic Vectors, MESH: Hematopoietic Stem Cell Transplantation, MESH: Lentivirus / genetics, MESH: Treatment Outcome, MESH: Wiskott-Aldrich Syndrome / genetics, MESH: Wiskott-Aldrich Syndrome / immunology, MESH: Wiskott-Aldrich Syndrome / therapy, [SDV.MHEP.PED]Life Sciences [q-bio]/Human health and pathology/Pediatrics, [SDV.BIO]Life Sciences [q-bio]/Biotechnology, [SDV.IMM]Life Sciences [q-bio]/Immunology
Relation: info:eu-repo/semantics/altIdentifier/pmid/35075289; info:eu-repo/grantAgreement//693762/EU/GENEFORCURE/GENEFORCURE; PUBMED: 35075289; PUBMEDCENTRAL: PMC8799465
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2Academic Journal
المؤلفون: Gougeon, Marie-Lise, Poirier-Beaudouin, Béatrice, Ausseil, Jérome, Zérah, Michel, Artaud, Cécile, Heard, Jean-Michel, Deiva, Kumaran, Tardieu, Marc
المساهمون: Département Infection et Epidémiologie - Department of Infection and Epidemiology, Institut Pasteur Paris (IP), Institut Toulousain des Maladies Infectieuses et Inflammatoires (Infinity), Université Toulouse III - Paul Sabatier (UT3), Université de Toulouse (UT)-Université de Toulouse (UT)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Institut Fédératif de Biologie (IFB), Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Service de neurochirurgie pédiatrique CHU Necker, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Necker - Enfants Malades AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Imagine - Institut des maladies génétiques (IHU) (Imagine - U1163), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité), Institut du Cerveau = Paris Brain Institute (ICM), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière AP-HP, Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Centre de Recherche Translationnelle - Center for Translational Science (CRT), Biothérapies pour les Maladies Neurodégénératives, Institut Pasteur Paris (IP)-Institut National de la Santé et de la Recherche Médicale (INSERM), Infectious Diseases Models for Innovative Therapies (IDMIT), Université Paris-Saclay-Institut de Biologie François JACOB (JACOB), Direction de Recherche Fondamentale (CEA) (DRF (CEA)), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Direction de Recherche Fondamentale (CEA) (DRF (CEA)), Commissariat à l'énergie atomique et aux énergies alternatives (CEA)-Commissariat à l'énergie atomique et aux énergies alternatives (CEA), Center for Immunology of Viral Infections and Autoimmune Diseases, Le Kremlin Bicêtre, Institut Pasteur sponsored the study during thefirst 30 monthsand UniQure sponsored it for the following 36 months. Thestudy was supported by funds from the“Association Française Contre les Myopathies (AFM)”, the patients‘association“Vaincreles Maladies Lysosomales”, the Institut Pasteur, the Conny MaevaCharitable Foundation, and a gift from the Akbaraly family.
المصدر: ISSN: 1664-3224.
مصطلحات موضوعية: AAV, CNS, MPS IIIB, NAGLU, T cells, cellular immunity, cytokines, MESH: Acetylglucosaminidase / genetics, MESH: Acetylglucosaminidase / immunology, MESH: Immunologic Memory, MESH: Lymphocyte Activation, MESH: Mucopolysaccharidosis III / complications, MESH: Mucopolysaccharidosis III / genetics, MESH: Child, MESH: Cytokines / metabolism, MESH: Drug Administration Routes, MESH: Genetic Therapy / adverse effects, MESH: Genetic Therapy / methods, MESH: Genetic Vectors / administration & dosage, MESH: Genetic Vectors / genetics, MESH: Immunity, Cellular, [SDV]Life Sciences [q-bio], [SDV.IMM]Life Sciences [q-bio]/Immunology
Relation: info:eu-repo/semantics/altIdentifier/pmid/34040605; PUBMED: 34040605; PUBMEDCENTRAL: PMC8141743
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3Academic Journal
المؤلفون: Mosbach, Valentine, Poggi, Lucie, Richard, Guy-Franck
المساهمون: Génétique des génomes - Genetics of Genomes (UMR 3525), Institut Pasteur Paris (IP)-Centre National de la Recherche Scientifique (CNRS), Collège Doctoral, Sorbonne Université (SU), SANOFI Recherche, The authors wish to thank the continuous support of the Institut Pasteur and of the Centre National de la Recherche Scientifique (CNRS). L. P. is the recipient of a Cifre PhD fellowship from Sanofi. V. M. was the recipient of two post-doctoral fellowships from Fondation Guy Nicolas and from Fondation Hardy.
المصدر: ISSN: 0172-8083.
مصطلحات موضوعية: Gene conversion, CRISPR-Cas9, Break-induced replication, ZFN, TALEN, Single-strand annealing, MESH: DNA / genetics, MESH: DNA / metabolism, MESH: Humans, MESH: Models, Genetic, MESH: Trinucleotide Repeat Expansion / genetics, MESH: Trinucleotide Repeats / genetics, MESH: DNA Breaks, Double-Stranded, MESH: DNA Repair, MESH: Endonucleases / metabolism, MESH: Genetic Therapy / methods, MESH: Genetic Therapy / trends, MESH: Heredodegenerative Disorders, Nervous System / genetics, Nervous System / metabolism, [SDV]Life Sciences [q-bio]
Relation: info:eu-repo/semantics/altIdentifier/pmid/29974202; pasteur-02864590; https://pasteur.hal.science/pasteur-02864590; https://pasteur.hal.science/pasteur-02864590/document; https://pasteur.hal.science/pasteur-02864590/file/CUGE-D-18-00094_V4.pdf; PUBMED: 29974202
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4Academic Journal
المؤلفون: Motta*, Jean-Paul, Magne*, Laurent, Descamps, Delphyne, Rolland, Corinne, Squarzoni-Dale, Camila, Rousset, Perrine, Balloy, Viviane, Huerre, Michel, Jenne, Dieter, Wartelle, Julien, Belaaouaj, Azzaq, Mas, Emmanuel, Vinel, Jean-Pierre, Alric, Laurent, Chignard, Michel, Vergnolle, Nathalie, Sallenave*, Jean-Michel
المساهمون: Centre de Physiopathologie Toulouse Purpan (CPTP), Université Toulouse III - Paul Sabatier (UT3), Université de Toulouse (UT)-Université de Toulouse (UT)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Défense innée et inflammation, Institut Pasteur Paris (IP)-Institut National de la Santé et de la Recherche Médicale (INSERM), Histotechnologie et Pathologie, Institut Pasteur Paris (IP), Department of Neuroimmunology, Max-Planck-Institut, Inflammation et immunité de l'épithélium respiratoire, Université de Reims Champagne-Ardenne (URCA)-IFR53, Service Gastroentérologie, hépatologie nutrition, diabétologie et maladies héréditaires du métabolisme pédiatrique CHU Toulouse, Pôle Enfants CHU Toulouse, Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Pôle Maladies de l'appareil digestif CHU Toulouse, Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Research was supported by grants from the Bettencourt-Schueller Foundation and the INSERM-Avenir (to N.V.), the Schlumberger Foundation (to N.V.), the Agence Nationale pour la Recherche (to N.V.), the Fondation pour la Recherche Medicale (to N.V.), the region Midi-Pyrenées (to J.-P.M.), and the Canadian Association of Gastroenterology (to C.S.-D.). Laurent Magne holds an ATER (temporary teaching fellow) position from the Université Paris 7-Diderot., The authors are indebted to Ms B. Solhonne (Institut Pasteur/INSERM U874, Paris, France), Ms M.-A. Nicola (Institut Pasteur, Paris), and to Professor Z. Xing (McMaster University, Hamilton, Canada) for stimulating discussions and help during the study.
المصدر: ISSN: 0016-5085.
مصطلحات موضوعية: Inflammation, Inflammatory Bowel Disease, Colitis, Proteases, Protease Inhibitors, Elafin, MESH: Adenoviridae/genetics, MESH: Animals, MESH: Gene Expression/physiology, MESH: Genetic Therapy/methods, MESH: HT29 Cells, MESH: Humans, MESH: Leukocyte Elastase/metabolism, MESH: Male, MESH: Mice, Inbred C57BL, Inbred CBA, Transgenic, MESH: Caco-2 Cells, MESH: Myeloblastin/metabolism, MESH: NF-kappa B/metabolism, MESH: Neutrophils/enzymology, MESH: Neutrophils/immunology, MESH: Protease Inhibitors/metabolism, MESH: Serine Proteinase Inhibitors/metabolism, MESH: Chemokines/metabolism, MESH: Colitis*/genetics, MESH: Colitis*/metabolism, MESH: Colitis*/therapy, MESH: Cytokines/metabolism
Relation: info:eu-repo/semantics/altIdentifier/pmid/21199654; pasteur-00552947; https://pasteur.hal.science/pasteur-00552947; https://pasteur.hal.science/pasteur-00552947/document; https://pasteur.hal.science/pasteur-00552947/file/Motta_et_al_Oct._2010_last_unmarked.pdf; PRODINRA: 404701; PUBMED: 21199654; WOS: 000288789900034
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5
المؤلفون: Valentine Mosbach, Lucie Poggi, Guy-Franck Richard
المساهمون: Génétique des génomes - Genetics of Genomes (UMR 3525), Institut Pasteur [Paris] (IP)-Centre National de la Recherche Scientifique (CNRS), Collège Doctoral, Sorbonne Université (SU), SANOFI Recherche, The authors wish to thank the continuous support of the Institut Pasteur and of the Centre National de la Recherche Scientifique (CNRS). L. P. is the recipient of a Cifre PhD fellowship from Sanofi. V. M. was the recipient of two post-doctoral fellowships from Fondation Guy Nicolas and from Fondation Hardy., Institut Pasteur [Paris]-Centre National de la Recherche Scientifique (CNRS), Collège doctoral [Sorbonne universités]
المصدر: Current Genetics
Current Genetics, 2019, 65, pp.17-28. ⟨10.1007/s00294-018-0865-1⟩
Current Genetics, Springer Verlag, 2019, 65, pp.17-28. ⟨10.1007/s00294-018-0865-1⟩مصطلحات موضوعية: MESH: Genetic Therapy / methods, DNA Repair, MESH: Genetic Therapy / trends, DNA repair, [SDV]Life Sciences [q-bio], Biology, MESH: DNA / genetics, MESH: DNA Repair, 03 medical and health sciences, MESH: Heredodegenerative Disorders, Nervous System / genetics, Trinucleotide Repeats, TALEN, MESH: Endonucleases / metabolism, MESH: Trinucleotide Repeat Expansion / genetics, MESH: Heredodegenerative Disorders, Nervous System / metabolism, Genetics, MESH: DNA Breaks, Double-Stranded, CRISPR, Humans, MESH: DNA / metabolism, DNA Breaks, Double-Stranded, Gene conversion, MESH: Models, Genetic, ZFN, MESH: Trinucleotide Repeats / genetics, 030304 developmental biology, Single-strand annealing, 0303 health sciences, Transcription activator-like effector nuclease, MESH: Humans, Models, Genetic, 030302 biochemistry & molecular biology, Break-induced replication, General Medicine, DNA, Genetic Therapy, Endonucleases, Zinc finger nuclease, Double Strand Break Repair, Heredodegenerative Disorders, Nervous System, CRISPR-Cas9, Homologous recombination, Trinucleotide repeat expansion, Trinucleotide Repeat Expansion
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6
المؤلفون: Perrine Rousset, Michel Huerre, Leopold F. Fröhlich, Camila Squarzoni Dale, Jean-Michel Sallenave, Laurence Martin, Corinne Rolland, Julien Wartelle, Delphyne Descamps, Laurent Alric, Jean-Paul Motta, Dieter E. Jenne, Laurent Magne, Jean-Pierre Vinel, Emmanuel Mas, Michel Chignard, Viviane Balloy, Nicolas Cenac, Nathalie Vergnolle, Azzaq Belaaouaj
المساهمون: Centre de Physiopathologie Toulouse Purpan (CPTP), Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Défense innée et inflammation, Institut Pasteur [Paris]-Institut National de la Santé et de la Recherche Médicale (INSERM), Histotechnologie et Pathologie, Institut Pasteur [Paris], Department of Neuroimmunology, Max-Planck-Institut, Inflammation et immunité de l'épithélium respiratoire, Université de Reims Champagne-Ardenne (URCA)-IFR53, Division of Gastroenterology, Hepatology and Nutrition, Hôpital pour enfants de Toulouse, Service de Gastro-entérologie - Hépatologie [Purpan], CHU Toulouse [Toulouse]-Hôpital Purpan [Toulouse], CHU Toulouse [Toulouse], Research was supported by grants from the Bettencourt-Schueller Foundation and the INSERM-Avenir (to N.V.), the Schlumberger Foundation (to N.V.), the Agence Nationale pour la Recherche (to N.V.), the Fondation pour la Recherche Medicale (to N.V.), the region Midi-Pyrenées (to J.-P.M.), and the Canadian Association of Gastroenterology (to C.S.-D.). Laurent Magne holds an ATER (temporary teaching fellow) position from the Université Paris 7-Diderot., The authors are indebted to Ms B. Solhonne (Institut Pasteur/INSERM U874, Paris, France), Ms M.-A. Nicola (Institut Pasteur, Paris), and to Professor Z. Xing (McMaster University, Hamilton, Canada) for stimulating discussions and help during the study., Centre de Physiopathologie Toulouse Purpan (ex IFR 30 et IFR 150) (CPTP), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Toulouse III - Paul Sabatier (UT3), Université Fédérale Toulouse Midi-Pyrénées-Université Fédérale Toulouse Midi-Pyrénées-Centre National de la Recherche Scientifique (CNRS), UFR Sciences du Vivant [Paris] (SDV), Université Paris Diderot - Paris 7 (UPD7), Institute of Pathology [Graz, Austria], University of Graz, Department of Neuroimmunology [Munich, Germany], Max Planck Institute of Neurobiology, Pôle Digestif [purpan], Hôpital Purpan [Toulouse], CHU Toulouse [Toulouse]-CHU Toulouse [Toulouse], Research was supported by grants from the Bettencourt-Schueller Foundation and the INSERM-Avenir (to N.V.), the Schlumberger Foundation (to N.V.), the Agence Nationale pour la Recherche (to N.V.), the Fondation pour la Recherche Medicale (to N.V.), the region Midi-Pyrenées (to J.-P.M.) ,and the Canadian Association of Gastroenterology (to C.S.-D.). Laurent Magne holds an ATER (temporary teaching fellow) position from the Université Paris7-Diderot., Université de Toulouse (UT)-Université de Toulouse (UT)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Institut Pasteur [Paris] (IP)-Institut National de la Santé et de la Recherche Médicale (INSERM), Institut Pasteur [Paris] (IP), Service Gastroentérologie, hépatologie nutrition, diabétologie et maladies héréditaires du métabolisme pédiatrique [CHU Toulouse], Pôle Enfants [CHU Toulouse], Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Pôle Maladies de l'appareil digestif [CHU Toulouse], Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Sallenave, Jean-Michel
المصدر: Gastroenterology
Gastroenterology, WB Saunders, 2010, 140 (4), pp.1272-82. ⟨10.1053/j.gastro.2010.12.050⟩
Gastroenterology, WB Saunders, 2011, 140 (4), pp.1272-1282. ⟨10.1053/j.gastro.2010.12.050⟩
Gastroenterology, 2010, 140 (4), pp.1272-82. ⟨10.1053/j.gastro.2010.12.050⟩مصطلحات موضوعية: MESH: Protease Inhibitors/metabolism, Male, Neutrophils, medicine.medical_treatment, [SDV]Life Sciences [q-bio], MESH: HT29 Cells, Gene Expression, MESH: Adenoviridae/genetics, Inflammatory bowel disease, MESH: Colitis*/genetics, Mice, 0302 clinical medicine, MESH: Genetic Therapy/methods, MESH: Leukocyte Elastase/metabolism, MESH: Colitis*/metabolism, MESH: Animals, MESH: Chemokines/metabolism, 0303 health sciences, MESH: Mice, Inbred CBA, biology, MESH: Elafin/genetics, Chemistry, Elastase, Gastroenterology, NF-kappa B, Proteases, Colitis, 3. Good health, Elafin, Neutrophil elastase, MESH: Neutrophils/enzymology, [SDV.IMM]Life Sciences [q-bio]/Immunology, Cytokines, 030211 gastroenterology & hepatology, MESH: Caco-2 Cells, MESH: NF-kappa B/metabolism, Chemokines, HT29 Cells, Serine Proteinase Inhibitors, [SDV.IMM] Life Sciences [q-bio]/Immunology, MESH: Mice, Transgenic, Myeloblastin, Mice, Transgenic, Protein degradation, MESH: Elafin/metabolism, Adenoviridae, 03 medical and health sciences, MESH: Serine Proteinase Inhibitors/metabolism, MESH: Mice, Inbred C57BL, medicine, Animals, Humans, Protease Inhibitors, MESH: Gene Expression/physiology, MESH: Mice, 030304 developmental biology, Inflammation, Protease, MESH: Humans, Hepatology, Inflammatory Bowel Disease, [SDV.MHEP.HEG]Life Sciences [q-bio]/Human health and pathology/Hépatology and Gastroenterology, Genetic Therapy, medicine.disease, digestive system diseases, MESH: Male, Mice, Inbred C57BL, MESH: Myeloblastin/metabolism, MESH: Colitis*/therapy, Immunology, MESH: Neutrophils/immunology, biology.protein, Cancer research, Mice, Inbred CBA, MESH: Cytokines/metabolism, Caco-2 Cells, Leukocyte Elastase
وصف الملف: application/pdf