المؤلفون: Liron Elkouby, Sean M. Armour, Raffaella Toso, Marti DiPietro, Robert J. Davidson, Giang N. Nguyen, Mallory Willet, Stephanie Kutza, Joseph Silverberg, Jennifer Frick, Marco Crosariol, Yuhuan Wang, Chuansong Wang, Katherine A. High, Denise E. Sabatino, Xavier M. Anguela

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 24, Iss , Pp 20-29 (2022)

مصطلحات موضوعية: hemophilia A, AAV, optimized vectors, codon optimization, low AAV dose, preclinical development, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050121001777; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/8d15f9b5838e44d49c75621a886fa6f5

المؤلفون: Helena Costa-Verdera, Fanny Collaud, Christopher R. Riling, Pauline Sellier, Jayme M. L. Nordin, G. Michael Preston, Umut Cagin, Julien Fabregue, Simon Barral, Maryse Moya-Nilges, Jacomina Krijnse-Locker, Laetitia van Wittenberghe, Natalie Daniele, Bernard Gjata, Jeremie Cosette, Catalina Abad, Marcelo Simon-Sola, Severine Charles, Mathew Li, Marco Crosariol, Tom Antrilli, William J. Quinn, David A. Gross, Olivier Boyer, Xavier M. Anguela, Sean M. Armour, Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi

المصدر: Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)

مصطلحات موضوعية: Science

وصف الملف: electronic resource

Relation: https://doaj.org/toc/2041-1723

URL الوصول: https://doaj.org/article/ecdd7a66d5fe4b0e825ff6e616f835cf

المؤلفون: Chenhui Zou, Koen O.A. Vercauteren, Eleftherios Michailidis, Mohammad Kabbani, Irene Zoluthkin, Corrine Quirk, Luis Chiriboga, Mustafa Yazicioglu, Xavier M. Anguela, Philip Meuleman, Katherine A. High, Roland W. Herzog, Ype P. de Jong

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 18, Iss , Pp 189-198 (2020)

مصطلحات موضوعية: humanized mice, AAV, gene therapy, liver, human hepatocytes, donor variability, Genetics, QH426-470, Cytology, QH573-671

وصف الملف: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050120301200; https://doaj.org/toc/2329-0501

URL الوصول: https://doaj.org/article/76e8a94fb75547cf982881d7ea650537

المؤلفون: Sean M. Armour, Mallory Willet, Marti A. DiPietro, Marco Crosariol, Stephanie Kutza, Raffaella Toso, Chuansong Wang, Katherine A. High, Robert J. Davidson, Jennifer Frick, Xavier M. Anguela, Liron Elkouby, Yuhuan Wang, Giang N. Nguyen, Denise E. Sabatino, Joseph Silverberg

المصدر: Molecular Therapy: Methods & Clinical Development, Vol 24, Iss, Pp 20-29 (2022)
Molecular Therapy. Methods & Clinical Development

مصطلحات موضوعية: optimized vectors, viruses, Genetic enhancement, Transgene, QH426-470, codon optimization, Immune system, Genetics, Potency, Medicine, Vector (molecular biology), Molecular Biology, Gene, QH573-671, business.industry, AAV, Orders of magnitude (mass), low AAV dose, Capsid, Immunology, Molecular Medicine, Original Article, hemophilia A, Cytology, business, preclinical development

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a46d008ab8ca27b5957160e1cc9ae294
https://doi.org/10.1016/j.omtm.2021.11.005

المؤلفون: Xavier M Anguela, Kristen Jaworski, Stacy E Croteau, John E J Rasko, Tiffany Chang, Federico Mingozzi, Paul E Monahan, Katherine A. High, Kathleen Z Reape, Margaret V Ragni, Lindsey A. George, Amy Macdougall, Spencer K. Sullivan, M Elaine Eyster, Benjamin J. Samelson-Jones, Robert Noble, Michael Recht, Marla Curran, Klaudia Kuranda

المصدر: N Engl J Med

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Adolescent, Genotype, Genetic enhancement, Genetic Vectors, Hemophilia A, Gastroenterology, Article, Young Adult, Immune system, Internal medicine, medicine, Humans, Vector (molecular biology), Adverse effect, Glucocorticoids, Immunosuppression Therapy, Lung, Factor VIII, business.industry, General Medicine, Genetic Therapy, Dependovirus, Middle Aged, Confidence interval, Discontinuation, medicine.anatomical_structure, Cohort, Hepatocytes, business, Follow-Up Studies

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a13db718b1db9c4d1a4f5c07a1a3abc2
https://pubmed.ncbi.nlm.nih.gov/34788507

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

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9a1664cfde76136fe6521768528975a1
https://hal.sorbonne-universite.fr/hal-03449262/document

المؤلفون: Mohammad Kabbani, Philip Meuleman, Eleftherios Michailidis, Ype P. de Jong, Corrine Quirk, Chenhui Zou, Luis Chiriboga, Katherine A. High, Mustafa N. Yazicioglu, Koen Vercauteren, Roland W. Herzog, Xavier M. Anguela, Irene Zoluthkin

المصدر: Molecular Therapy. Methods & Clinical Development
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
Molecular Therapy: Methods & Clinical Development, Vol 18, Iss, Pp 189-198 (2020)

مصطلحات موضوعية: 0301 basic medicine, lcsh:QH426-470, Genetic enhancement, Transgene, Biology, liver, GENE-TRANSFER, Virus, Article, Green fluorescent protein, 03 medical and health sciences, Transduction (genetics), Chimera (genetics), 0302 clinical medicine, hemophilia, Medicine and Health Sciences, Genetics, medicine, lcsh:QH573-671, Molecular Biology, Gene, IN-VIVO, lcsh:Cytology, human hepatocytes, donor variability, AAV, EXPANSION, HEPATIC-DYSFUNCTION, gene therapy, Cell biology, lcsh:Genetics, ENGRAFTMENT, 030104 developmental biology, medicine.anatomical_structure, humanized mice, ADENOASSOCIATED VIRUS, MURINE LIVER, 030220 oncology & carcinogenesis, Hepatocyte, REPLICATION, VECTORS, Molecular Medicine, C VIRUS-INFECTION

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::425ecf07b20f36a97b45039f547d4ad7
http://europepmc.org/articles/PMC7326722

المؤلفون: Xavier M. Anguela, Katherine A. High

المصدر: Annual Review of Medicine. 70:273-288

مصطلحات موضوعية: Male, 0301 basic medicine, Genetic enhancement, Genetic Vectors, Bioinformatics, Risk Assessment, General Biochemistry, Genetics and Molecular Biology, Adenoviridae, Translational Research, Biomedical, 03 medical and health sciences, 0302 clinical medicine, Momentum (finance), Animals, Humans, Medicine, Gene Editing, business.industry, Lentivirus, Genetic Therapy, General Medicine, United States, Treatment Outcome, 030104 developmental biology, National Institutes of Health (U.S.), 030220 oncology & carcinogenesis, Female, business, Forecasting

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5720c1d65c6e7827cdf0b1e2508d406d
https://doi.org/10.1146/annurev-med-012017-043332

المؤلفون: Margareth C. Ozelo, Katie Wachtel, Yifeng Chen, Benjamin J. Samelson-Jones, Leslie Raffini, Katherine A. High, Margaret V. Ragni, John E.J. Rasko, Xavier M. Anguela, Lindsey A. George, Jennifer Wellman, Alexa R. Runowski, Federico Mingozzi, Maria Hazbon, Klaudia Kuranda

المصدر: Mol Ther

مصطلحات موضوعية: Male, Genetic enhancement, viruses, medicine.disease_cause, Antibodies, Viral, Factor IX, 0302 clinical medicine, Drug Discovery, Vector (molecular biology), Longitudinal Studies, Neutralizing antibody, Adeno-associated virus, 0303 health sciences, biology, Gene Transfer Techniques, Dependovirus, Middle Aged, Treatment Outcome, Liver, 030220 oncology & carcinogenesis, Hepatocellular carcinoma, Molecular Medicine, Original Article, Antibody, Signal Transduction, Adult, Genetic Vectors, Cross Reactions, Hemophilia B, Virus, 03 medical and health sciences, Young Adult, Immune system, Capsid, Genetics, medicine, Humans, Infusions, Intra-Arterial, Molecular Biology, 030304 developmental biology, Pharmacology, business.industry, Genetic Therapy, medicine.disease, Antibodies, Neutralizing, Immunology, biology.protein, Hepatocytes, business, Follow-Up Studies

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4e4ea62b5161a331b808e5546cb819c4
https://pubmed.ncbi.nlm.nih.gov/32559433

المؤلفون: Adeline Miranda, Dan Lupo, Federico Mingozzi, Xavier M. Anguela, Joseph Silverberg, Karen Huang, Heena Beck, Saghana Muraleetharan, Béatrice Marolleau, Fanny Collaud, Laetitia van Wittengerghe, Sean M. Armour, Christian Leborgne, Elena Barbon, Sandrine Delignat, Jeffrey M. Alexander, Hayley Hanby, Victoria Daventure, Giuseppe Ronzitti, Sébastien Lacroix-Desmazes, Anna Fabiano, Daniel M. Cohen

المساهمون: 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, Spark Therapeutics [Philadelphia, PA, USA], Centre de Recherche des Cordeliers (CRC (UMR_S_1138 / U1138)), École pratique des hautes études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Université de Paris (UP), École Pratique des Hautes Études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Université Paris Cité (UPCité)

المصدر: Nature Medicine
Nature Medicine, Nature Publishing Group, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩
Nature Medicine, 2020, 26 (7), pp.1096-1101. ⟨10.1038/s41591-020-0911-7⟩

مصطلحات موضوعية: 0301 basic medicine, biology, business.industry, Genetic enhancement, medicine.medical_treatment, viruses, General Medicine, Immunotherapy, Virology, General Biochemistry, Genetics and Molecular Biology, In vitro, Virus, Endopeptidase, 3. Good health, 03 medical and health sciences, Transduction (genetics), 030104 developmental biology, 0302 clinical medicine, In vivo, 030220 oncology & carcinogenesis, biology.protein, Medicine, [SDV.IMM]Life Sciences [q-bio]/Immunology, Antibody, business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ff04724b6af379a7e9370adc25a9aa43
https://hal.archives-ouvertes.fr/hal-03031762