-
1دورية أكاديمية
المؤلفون: Jenny A. Greig, Roberto Calcedo, Leticia Kuri-Cervantes, Jayme M.L. Nordin, Jessica Albrecht, Erin Bote, Tamara Goode, Edward A. Chroscinski, Peter Bell, Laura K. Richman, Michael R. Betts, James M. Wilson
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 11, Iss , Pp 191-201 (2018)
وصف الملف: electronic resource
-
2دورية أكاديمية
المؤلفون: Jenny A. Greig, Jayme M.L. Nordin, Christine Draper, Deirdre McMenamin, Edward A. Chroscinski, Peter Bell, John T. Gray, Laura K. Richman, James M. Wilson
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 237-244 (2018)
وصف الملف: electronic resource
-
3
المؤلفون: Vincent H. Wu, Son Nguyen, Jayme M.L. Nordin, Jaimy Joy, Felicity Mampe, Pablo Tebas, Luis J. Montaner, Katharine J. Bar, Laura A. Vella, Michael R. Betts
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::b0facd7fc68349f93c5aa83dfb63aa63
https://doi.org/10.1101/2022.04.02.486753 -
4
المؤلفون: Catalina Abad, N. Danièle, Jayme M.L. Nordin, Giuseppe Ronzitti, Bernard Gjata, Helena Costa-Verdera, Simon Barral, Sean M. Armour, Marcelo Simon-Sola, Fanny Collaud, Marco Crosariol, Julien Fabregue, David A. Gross, Mathew Li, Jérémie Cosette, Laetitia van Wittenberghe, Pasqualina Colella, Maryse Moya-Nilges, G. Michael Preston, Christopher Riling, Xavier M. Anguela, Federico Mingozzi, Severine Charles, Pauline Sellier, Tom Antrilli, William J. Quinn, Umut Cagin, Jacomina Krijnse-Locker, Olivier Boyer
المساهمون: Généthon, Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE), Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris-Saclay-Généthon, Centre de recherche en Myologie – U974 SU-INSERM, Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU), Spark Therapeutics [Philadelphia, PA, USA], Institut Pasteur [Paris] (IP), Plateforme BioImagerie Ultrastructurale – Ultrastructural BioImaging Platform (UTechS UBI), Institute for Research and Innovation in Biomedicine (IRIB), Université de Rouen Normandie (UNIROUEN), Normandie Université (NU)-Normandie Université (NU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), This work was supported by Genethon and the French Muscular Dystrophy Association,the European Union’s research and innovation program under grant agreement no.667751 (to F.M.), the European Research Council Consolidator Grant under grantagreement no. 617432 (to F.M.), Marie Skłodowska-Curie Actions Individual Fellowship(MSCA-IF) grant agreement no. 797144 (to U.C.), a grant from the DIM ThérapieGénique (to D.A.G.) and by Spark Therapeutics under a sponsored research agreementto Genethon., ANR-16-CE92-0008,membrane dynamics,Rupture et réparation membranaire : stratégies d'assemblage virale(2016), European Project: 667751,H2020,H2020-PHC-2015-two-stage,MYOCURE(2016), European Project: 617432,EC:FP7:ERC,ERC-2013-CoG,MOMAAV(2014), Gestionnaire, Hal Sorbonne Université, Development of an innovative gene therapy platform to cure rare hereditary muscle disorders - MYOCURE - - H20202016-01-01 - 2019-12-31 - 667751 - VALID, Molecular signatures and Modulation of immunity to Adeno-Associated Virus vectors - MOMAAV - - EC:FP7:ERC2014-07-01 - 2019-06-30 - 617432 - VALID, Thérapie des maladies du muscle strié, Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Sorbonne Université (SU), Institut Pasteur [Paris]
المصدر: Nature Communications
Nature Communications, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩
Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)
Nature Communications, Nature Publishing Group, 2021, 12 (1), pp.6393. ⟨10.1038/s41467-021-26744-4⟩مصطلحات موضوعية: Male, congenital, hereditary, and neonatal diseases and abnormalities, Genetic enhancement, Science, Metabolic disorders, General Physics and Astronomy, Pharmacology, General Biochemistry, Genetics and Molecular Biology, Virus, Article, law.invention, 03 medical and health sciences, Mice, 0302 clinical medicine, Gene therapy, Pharmacokinetics, law, Autophagy, Distribution (pharmacology), Medicine, Animals, Enzyme Replacement Therapy, 030304 developmental biology, chemistry.chemical_classification, 0303 health sciences, Multidisciplinary, [SDV.MHEP] Life Sciences [q-bio]/Human health and pathology, business.industry, Glycogen Storage Disease Type II, nutritional and metabolic diseases, alpha-Glucosidases, General Chemistry, Enzyme replacement therapy, Phenotype, 3. Good health, Enzyme, chemistry, Liver, Recombinant DNA, Female, business, 030217 neurology & neurosurgery, [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology
وصف الملف: application/pdf
-
5
المؤلفون: Tamara Goode, Elizabeth L. Buza, Lisa M. Kattenhorn, Melanie K. Smith, Jayme M.L. Nordin, Samuel C. Wadsworth, Edward A. Chroscinski, James M. Wilson, Jenny A. Greig, Nicole Schmidt
المصدر: Human gene therapy. 33(7-8)
مصطلحات موضوعية: Male, Genetic Vectors, Physiology, Hemophilia A, Mice, Blood loss, Genetics, Medicine, Liver transaminases, Animals, Humans, Vector (molecular biology), Molecular Biology, Factor VIII, biology, business.industry, Tail vein, Genetic Therapy, Dependovirus, Effective dose (pharmacology), Disease Models, Animal, biology.protein, Molecular Medicine, Female, Antibody, Bilirubin levels, business, Serum chemistry
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1f3cf0d54c0720bede4234e43a9b2a9a
https://pubmed.ncbi.nlm.nih.gov/34652966 -
6
المؤلفون: Scott N. Ashley, Peter Bell, Elizabeth L. Buza, Jayme M.L. Nordin, Melanie K. Smith, Yanqing Zhu, Christine Draper, James M. Wilson, Jenny A. Greig
المصدر: Human Gene Therapy Clinical Development. 30:29-39
مصطلحات موضوعية: medicine.medical_specialty, Transgene, Genetic enhancement, ATPase, Mice, Transgenic, Disease, Mice, Liver disease, Hepatolenticular Degeneration, Internal medicine, Animals, Humans, Medicine, Vector (molecular biology), Gene, Genetics (clinical), biology, business.industry, Genetic Therapy, Dependovirus, Copper Chelation, medicine.disease, Disease Models, Animal, Endocrinology, Liver, Copper-Transporting ATPases, Mutation, biology.protein, business, Copper
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::fba4c33bd2962d13438e6d7756ebd430
https://doi.org/10.1089/humc.2018.219 -
7
المؤلفون: Jayme M.L. Nordin, Jenny A. Greig, Erin Bote, James M. Wilson, Peter Bell, Laura K. Richman, Roberto Calcedo, Leticia Kuri-Cervantes, Jessica Albrecht, Edward A. Chroscinski, Michael R. Betts, Tamara Goode
المصدر: Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 11, Iss, Pp 191-201 (2018)مصطلحات موضوعية: 0301 basic medicine, lcsh:QH426-470, Lymphocyte, Genetic enhancement, Transgene, T cell, Biology, liver, Article, immune response, Epitope, 03 medical and health sciences, Immune system, Genetics, medicine, Crigler-Najjar, lcsh:QH573-671, Molecular Biology, Ornithine transcarbamylase deficiency, lcsh:Cytology, AAV, medicine.disease, gene therapy, lcsh:Genetics, 030104 developmental biology, medicine.anatomical_structure, Immunology, Molecular Medicine, UGT1A1, transgene expression, CD8
-
8
المؤلفون: Laura K. Richman, Jayme M.L. Nordin, Peter Bell, Edward A. Chroscinski, James M. Wilson, Jenny A. Greig, Christine Draper, John T. Gray, Deirdre McMenamin
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 10, Iss, Pp 237-244 (2018)
Molecular Therapy. Methods & Clinical Developmentمصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, lcsh:QH426-470, Bilirubin, Crigler–Najjar syndrome, Genetic enhancement, adeno-associated virus, Pharmacology, liver, medicine.disease_cause, Article, 03 medical and health sciences, chemistry.chemical_compound, Genetics, medicine, lcsh:QH573-671, Molecular Biology, Adeno-associated virus, Crigler-Najjar Syndrome, Clinical pathology, lcsh:Cytology, business.industry, Jaundice, medicine.disease, Effective dose (pharmacology), lcsh:Genetics, 030104 developmental biology, chemistry, Knockout mouse, Molecular Medicine, bilirubin, medicine.symptom, business
-
9
المؤلفون: Jenny A. Greig, Christine Draper, James M. Wilson, Peter Bell, Jayme M.L. Nordin
المصدر: Human Gene Therapy. 29:763-770
مصطلحات موضوعية: 0301 basic medicine, Bilirubin, viruses, Genetic enhancement, Genetic Vectors, Mice, 03 medical and health sciences, chemistry.chemical_compound, Genetics, Animals, Humans, Medicine, Glucuronosyltransferase, Molecular Biology, Crigler-Najjar Syndrome, Mice, Knockout, business.industry, food and beverages, Genetic Therapy, Dependovirus, Phenotype, Disease Models, Animal, 030104 developmental biology, Animals, Newborn, Liver, chemistry, Cancer research, Molecular Medicine, business
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d9b53c3d2a393980ff6a6f41bc0f89fc
https://doi.org/10.1089/hum.2017.185 -
10
المؤلفون: Francesco Puzzo, Xavier M. Anguela, Pauline Sellier, Hayley Hanby, Christopher Riling, Federico Mingozzi, Giuseppe Ronzitti, Helena Costa Verdera, Daniel M. Cohen, William J. Quinn, Jayme M.L. Nordin, George M. Preston, Umut Cagin, Virginia Haurigot, Fanny Collaud, Sean M. Armour, Pasqualina Colella, Marco Crosariol
المصدر: Molecular Genetics and Metabolism. 129:S22-S23
مصطلحات موضوعية: Oncology, medicine.medical_specialty, Endocrinology, business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Internal medicine, Genetics, medicine, Disease, business, Molecular Biology, Biochemistry