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المؤلفون: Benjamin Arnson, Hye Ri Kang, Elizabeth D. Brooks, Dorothy Gheorghiu, Ekaterina Ilich, David Courtney, Jeffrey I. Everitt, Bryan R. Cullen, Dwight D. Koeberl
المصدر: Arnson, B, Kang, H R, Brooks, E D, Gheorghiu, D, Ilich, E, Courtney, D, Everitt, J I, Cullen, B R & Koeberl, D D 2023, ' Genome editing using Staphylococcus aureus Cas9 in a canine model of glycogen storage disease Ia ', Molecular Therapy: Methods and Clinical Development, vol. 29, pp. 108-119 . https://doi.org/10.1016/j.omtm.2023.03.001Test
Mol Ther Methods Clin Devمصطلحات موضوعية: glucose-6-phosphatase, glycogen storage disease, adeno-associated virus vectors, Genetics, genome editing, Molecular Medicine, Original Article, von Gierke disease, CRISPR-Cas9, inherited disorder of metabolism, Molecular Biology
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7395c40b4f90eb3a8998a5a2a74a00f2Test
https://doi.org/10.1016/j.omtm.2023.03.001Test -
2تقرير
المؤلفون: Ibraheim, Raed, Tai, Phillip W. L., Mir, Aamir, Javeed, Nida, Wang, Jiaming, Nelson, Samantha J., Khokhar, Eraj, Mintzer, Esther, Maitland, Stacy A., Cao, Yueying, Gao, Guangping, Sontheimer, Erik J.
المساهمون: Graduate School of Biomedical Sciences, Viral Vector Core, Li Weibo Institute for Rare Diseases Research, Program in Molecular Medicine, Department of Molecular, Cell and Cancer Biology, Department of Microbiology and Physiological Systems, Horae Gene Therapy Center, RNA Therapeutics Institute
المصدر: bioRxiv
مصطلحات موضوعية: Molecular Biology, Cas9, Genome Editing, virus vectors, therapeutics, Genetics and Genomics, Nucleic Acids, Nucleotides, and Nucleosides, Viruses
العلاقة: Now published in Nature Communications doi:10.1038/s41467-021-26518-y; bioRxiv 2020.10.09.333997; doi: https://doi.org/10.1101/2020.10.09.333997Test. Link to preprint on bioRxiv .; http://hdl.handle.net/20.500.14038/29595Test; https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=2838&context=faculty_pubs&unstamped=1Test; https://escholarship.umassmed.edu/faculty_pubs/1813Test; faculty_pubs/1813
الإتاحة: https://doi.org/10.1101/2020.10.09.333997Test
https://doi.org/20.500.14038/29595Test
https://doi.org/10.1038/s41467-021-26518-yTest
https://hdl.handle.net/20.500.14038/29595Test
https://escholarship.umassmed.edu/cgi/viewcontent.cgi?article=2838&context=faculty_pubs&unstamped=1Test
https://escholarship.umassmed.edu/faculty_pubs/1813Test -
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المؤلفون: Yuxiang Wang, Anthony Griffiths, Douglas E. Brackney, Paulo H. Verardi
المصدر: Pathogens; Volume 11; Issue 12; Pages: 1505
مصطلحات موضوعية: Microbiology (medical), Infectious Diseases, General Immunology and Microbiology, recombinant vaccinia virus vectors, arboviruses, virus-like particles, Powassan virus, Heartland virus, severe fever with thrombocytopenia syndrome virus, Bourbon virus, Mayaro virus, Immunology and Allergy, Molecular Biology
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ffacbb8453b2738171908b3d95fb9a51Test
https://doi.org/10.3390/pathogens11121505Test -
4رسالة جامعية
المؤلفون: Li, Wenjun
المساهمون: Li, Chengwen, Wallet, Shannon, Hirsch, Matthew, Loeser, Richard, Arnold, Roland
مصطلحات موضوعية: Immune Modulation, Adeno-Associated Virus Vectors, Biomedical engineering, Gene Therapy, Immunology, Molecular biology, Arthritis
العلاقة: https://doi.org/10.17615/p8kc-az55Test; https://cdr.lib.unc.edu/downloads/zk51vt95m?file=thumbnailTest; https://cdr.lib.unc.edu/downloads/zk51vt95mTest
الإتاحة: https://doi.org/10.17615/p8kc-az55Test
https://cdr.lib.unc.edu/downloads/zk51vt95m?file=thumbnailTest
https://cdr.lib.unc.edu/downloads/zk51vt95mTest -
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المؤلفون: Christina Wagner, Bernd Innthaler, Martin Lemmerer, Robert Pletzenauer, Ruth Birner-Gruenberger
المصدر: International Journal of Molecular Sciences; Volume 23; Issue 21; Pages: 12715
مصطلحات موضوعية: Light, Genetic Vectors, Organic Chemistry, General Medicine, adeno-associated virus vectors, ion-exchange chromatography, multi-angle light scattering, dynamic light scattering, protein characterization, critical quality attributes, Dependovirus, Chromatography, Ion Exchange, Catalysis, Computer Science Applications, Inorganic Chemistry, Chromatography, Gel, Humans, Capsid Proteins, Physical and Theoretical Chemistry, Molecular Biology, Chromatography, High Pressure Liquid, Spectroscopy
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::17f9bcfa2c1273173e2aeb2aca551d2fTest
https://doi.org/10.3390/ijms232112715Test -
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المؤلفون: Atsushi Miyanohara, Tracy Guo, Dimosthenis Giamouridis, Matthijs Blankesteijn, H. Kirk Hammond, Erik A.L. Biessen, Young Chul Kim, N. Chin Lai, Mei Hua Gao, Zhen Tan
المساهمون: Farmacologie en Toxicologie, RS: Carim - H03 ECM and Wnt signaling, RS: CARIM - R2 - Cardiac function and failure, RS: Carim - B07 The vulnerable plaque: makers and markers, Pathologie, RS: CARIM - R3 - Vascular biology
المصدر: Human Gene Therapy, 30(1), 10-20. Mary Ann Liebert Inc.
مصطلحات موضوعية: Male, PHARMACOKINETICS, medicine.medical_treatment, Gene Expression, Apoptosis, MOUSE, Sarcomere, Ventricular Function, Left, Mice, 0302 clinical medicine, Transduction, Genetic, Troponin I, Gene Order, Myocyte, FAILURE, Myocytes, Cardiac, Myocardial infarction, Transgenes, Saline, Urocortins, Research Articles, Urocortin, 0303 health sciences, Ejection fraction, Gene Transfer Techniques, Dependovirus, MUSCLE, gene therapy, Echocardiography, 030220 oncology & carcinogenesis, Cardiology, Molecular Medicine, Female, AAV8, ADENOASSOCIATED VIRUS VECTORS, EXPRESSION, medicine.medical_specialty, Genetic Vectors, 03 medical and health sciences, Internal medicine, heart failure with reduced EF, Genetics, medicine, Animals, Molecular Biology, 030304 developmental biology, Heart Failure, business.industry, Genetic Therapy, medicine.disease, Fibrosis, DYSFUNCTION, Disease Models, Animal, MYOCARDIAL-INFARCTION, Heart failure, Calcium, business, SERCA2a, Biomarkers
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3889b3fe9318d54cd5b449cd104d72bcTest
https://cris.maastrichtuniversity.nl/en/publications/4d752fe3-a7a2-4f49-bb2d-1586f12fdb4cTest -
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المؤلفون: Nicole Armbruster, Jennifer Krieg, Manuel Weißenberger, Carsten Scheller, Andre F. Steinert
المصدر: Frontiers in Pharmacology
Frontiers in Pharmacology, Vol 8 (2017)مصطلحات موضوعية: 0301 basic medicine, foamy virus, Transgene, pellet culture, Biology, Green fluorescent protein, Flow cytometry, 03 medical and health sciences, chondrogenesis, medicine, Pharmacology (medical), mesenchymal stem cell, Original Research, Pharmacology, IL1RA, medicine.diagnostic_test, ddc:617, Mesenchymal stem cell, lcsh:RM1-950, Interleukin, virus vectors, Chondrogenesis, Molecular biology, interleukin 1 receptor antagonist, 030104 developmental biology, Interleukin 1 receptor antagonist, lcsh:Therapeutics. Pharmacology, arthritis, Cell culture
وصف الملف: application/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c7dee5ae07d32202824aecb1cb5f4c46Test
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المؤلفون: Yifeng Chen, F Mingozzi, Shyrie C Edmonson, Margriet J. Vervoordeldonk, Rogier M. Thurlings, Katherine A. High, Paul P. Tak, Shangzhen Zhou
المساهمون: Clinical Immunology and Rheumatology
المصدر: Gene therapy, 20(4), 417-424. Nature Publishing Group
Gene Therapyمصطلحات موضوعية: viruses, Genetic Vectors, Arthritis, Biology, Arthritis, Rheumatoid, Antibodies, Monoclonal, Murine-Derived, 03 medical and health sciences, 0302 clinical medicine, Transduction, Genetic, Immunity, Synovial Fluid, Genetics, medicine, Humans, Synovial fluid, neutralizing antibodies, Neutralizing antibody, Molecular Biology, 030304 developmental biology, B-Lymphocytes, synovial tissue, 0303 health sciences, Genetic Therapy, Dependovirus, medicine.disease, Antibodies, Neutralizing, Virology, Immunity, Humoral, 3. Good health, Titer, 030220 oncology & carcinogenesis, Rheumatoid arthritis, Humoral immunity, Immunology, adeno-associated virus vectors, biology.protein, Molecular Medicine, Original Article, Immunotherapy, Antibody, Rituximab
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4917cafc37ec063a9e003bdb27690784Test
https://doi.org/10.1038/gt.2012.55Test -
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المؤلفون: Bernard L. Schneider, Patrick Aebischer, Veronica Setola, Chris Towne
المصدر: Molecular Therapy. 19(2):274-283
مصطلحات موضوعية: Transgene, Genetic Vectors, Mice, Transgenic, Gene delivery, Biology, Adenoassociated Virus Vectors, Injections, Intramuscular, Neuroprotection, Mice, Superoxide Dismutase-1, Drug Discovery, medicine, Genetics, Animals, Humans, Gene silencing, RNA, Small Interfering, Amyotrophic lateral sclerosis, Transgenic Als Mice, Molecular Biology, Prolongs Survival, Motor Neurons, Rna Interference, Pharmacology, Gene knockdown, Skeletal-Muscle, Mouse Model, Superoxide Dismutase, Amyotrophic Lateral Sclerosis, Amyotrophic-Lateral-Sclerosis, Genetic Therapy, Dependovirus, Motor neuron, medicine.disease, Molecular biology, Muscle atrophy, Cell biology, Disease Models, Animal, medicine.anatomical_structure, Disease Progression, Degeneration, Molecular Medicine, Original Article, medicine.symptom, Delivery
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f2873a31022371dba2c2eef49d84c87bTest
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المؤلفون: Alexandros Katoulis, Ronald A. Li, Fotis Kolokathis, Roger J. Hajjar, Evangelia G. Kranias, Chi Wing Kong, Francesca Stillitano, Folkert W. Asselbergs, Dimitrios Tsiapras, Johannes M.I.H. Gho, Despina Sanoudou, Joseph C. Wu, Jean-Sébastien Hulot, Aryan Vink, Mathieu Nonnenmacher, Delaine K. Ceholski, Jens Hansen, Ioannis Karakikes, Lihuan Ren, Christos Tzimas, Dimitrios Th. Kremastinos, Vittavat Termglinchan, Ninette Cohen, Stephanie N. Rushing
المساهمون: Icahn School of Medicine at Mount Sinai [New York] (MSSM), Stanford School of Medicine [Stanford], Stanford Medicine, Stanford University-Stanford University, Biomedical Research Foundation of the Academy of Athens, National and Kapodistrian University of Athens (NKUA), LKS Faculty of Medicine (HKU Med), The University of Hong Kong (HKU), University Medical Center [Utrecht], Onassis Cardiac Surgery Center [Athens] (OCSC), ICIN - Netherlands Heart Institute, University College of London [London] (UCL), Unité de Recherche sur les Maladies Cardiovasculaires, du Métabolisme et de la Nutrition = Institute of cardiometabolism and nutrition (ICAN), Université Pierre et Marie Curie - Paris 6 (UPMC)-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], Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), University of Cincinnati (UC), HAL UPMC, Gestionnaire, Unité de Recherche sur les Maladies Cardiovasculaires, du Métabolisme et de la Nutrition = Research Unit on Cardiovascular and Metabolic Diseases (ICAN), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU)
المصدر: Nature Communications
Nature Communications, Nature Publishing Group, 2015, 6, pp.6955. ⟨10.1038/ncomms7955⟩
Nature Communications, 2015, 6, pp.6955. ⟨10.1038/ncomms7955⟩
Nature Communications [E], 6. Nature Publishing Groupمصطلحات موضوعية: Cardiomyopathy, General Physics and Astronomy, 030204 cardiovascular system & hematology, ARRHYTHMOGENIC CARDIOMYOPATHY, CARDIOMYOCYTES, DISEASE, 0302 clinical medicine, Myocytes, Cardiac, Induced pluripotent stem cell, Sequence Deletion, 0303 health sciences, Transcription activator-like effector nuclease, Multidisciplinary, Deoxyribonucleases, [SDV.MHEP] Life Sciences [q-bio]/Human health and pathology, HYPERTROPHIC CARDIOMYOPATHY, Targeted Gene Repair, DIRECTED DIFFERENTIATION, Gene Transfer Techniques, Phenotype, 3. Good health, Phospholamban, Female, Cardiomyopathies, PLURIPOTENT STEM-CELLS, ADENOASSOCIATED VIRUS VECTORS, Adult, endocrine system, Induced Pluripotent Stem Cells, LONG-QT SYNDROME, Biology, General Biochemistry, Genetics and Molecular Biology, Article, Adenoviridae, 03 medical and health sciences, Directed differentiation, medicine, Humans, Gene, 030304 developmental biology, Calcium-Binding Proteins, General Chemistry, IN-VITRO, medicine.disease, DILATED CARDIOMYOPATHY, Molecular biology, Cancer research, [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology
وصف الملف: application/pdf; image/pdf
الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7337ab9cafe0bbdb88fdddd8cfd5c7b6Test
https://hal.sorbonne-universite.fr/hal-01292425/documentTest