-
1
المؤلفون: Joseph E. Kaserman, Lauren Young, Andrew A. Wilson, Yvonne Aratyn-Schaus, Packer Michael, Carlos Villacorta-Martin, Gregoire Francine, Jonathan Lindstrom-Vautrin, Rhiannon B Werder
المصدر: Molecular Therapy. 29:3219-3229
مصطلحات موضوعية: Induced Pluripotent Stem Cells, Gene Expression, Alpha (ethology), medicine.disease_cause, alpha 1-Antitrypsin Deficiency, Drug Discovery, Genetics, medicine, Humans, Secretion, Induced pluripotent stem cell, Molecular Biology, Cells, Cultured, Gene Editing, Pharmacology, Mutation, Alpha 1-antitrypsin deficiency, Chemistry, Adenine, RNA, Cell Differentiation, Endoplasmic Reticulum Stress, medicine.disease, Molecular biology, medicine.anatomical_structure, alpha 1-Antitrypsin, Hepatocyte, Hepatocytes, Unfolded protein response, Molecular Medicine, CRISPR-Cas Systems, Biomarkers
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::30a607aaccda9c5ddc82338a01c7fef4
https://doi.org/10.1016/j.ymthe.2021.06.021 -
2
المؤلفون: Ewa Janosz, Falk F. R. Buettner, Nico Lachmann, Axel Schambach, Marc Schwabbauer, Thomas Moritz, Constantin von Kaisenberg, Doreen Kloos, Miriam Hetzel, Hanna Spielmann, Charlotte Rossdam, Srinu Tumpara, Sabina Janciauskiene
المصدر: Gene Therapy
مصطلحات موضوعية: 0301 basic medicine, congenital, hereditary, and neonatal diseases and abnormalities, Transgene, Genetic enhancement, CD11c, Genetic transduction, Biology, Cell delivery, Animals, Genetically Modified, Mice, 03 medical and health sciences, Gene therapy, 0302 clinical medicine, In vivo, alpha 1-Antitrypsin Deficiency, Genetics, medicine, Animals, Humans, Lung, Molecular Biology, Respiratory tract diseases, medicine.diagnostic_test, Haematopoietic stem cells, Macrophages, Molecular biology, Transplantation, 030104 developmental biology, Bronchoalveolar lavage, medicine.anatomical_structure, Pulmonary Emphysema, Perspective, Molecular Medicine, 030217 neurology & neurosurgery
-
3
المؤلفون: Michael S. Packer, Vivek Chowdhary, Genesis Lung, Lo-I Cheng, Yvonne Aratyn-Schaus, Dominique Leboeuf, Sarah Smith, Aalok Shah, Delai Chen, Marina Zieger, Brian J. Cafferty, Bo Yan, Giuseppe Ciaramella, Francine M. Gregoire, Christian Mueller
المصدر: Mol Ther
مصطلحات موضوعية: Pharmacology, Gene Editing, Adenine, Cytosine, Mice, alpha 1-Antitrypsin, alpha 1-Antitrypsin Deficiency, Drug Discovery, Liposomes, Mutation, Genetics, Molecular Medicine, Animals, Humans, Nanoparticles, Original Article, Molecular Biology
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::36644562d320c3f61f413b126663bc6b
https://pubmed.ncbi.nlm.nih.gov/35121111 -
4
المؤلفون: Liang Chen, Jia‑Yong Zhong, Jun Bai, Wang Liao, Xian‑Jin Wu, Shuang‑Ping Liu, Ruo Hu
المصدر: Molecular Medicine Reports
مصطلحات موضوعية: 0301 basic medicine, Proteomics, Cancer Research, Apolipoprotein B, Biochemistry, 0302 clinical medicine, subcutaneous immunotherapy, Gene Regulatory Networks, Child, biology, Pyroglyphidae, dust mite-related asthma, Dust, Articles, isobaric tags for relative and absolute quantification, Treatment Outcome, Oncology, 030220 oncology & carcinogenesis, Child, Preschool, Proteome, Apolipoprotein B-100, Molecular Medicine, Female, Allergen immunotherapy, Injections, Subcutaneous, Antithrombin III, 03 medical and health sciences, Genetics, medicine, Mite, Animals, Humans, Molecular Biology, Asthma, House dust mite, Oncogene, business.industry, Computational Biology, medicine.disease, biology.organism_classification, Molecular medicine, Fibronectins, 030104 developmental biology, Gene Expression Regulation, Desensitization, Immunologic, Case-Control Studies, alpha 1-Antitrypsin, Immunology, biology.protein, business, Keratin-1, Biomarkers
-
5
المؤلفون: Amber Vu, Paul B. McCray
المصدر: Hum Gene Ther
مصطلحات موضوعية: Cystic Fibrosis, Genetic enhancement, Genetic Vectors, Cystic Fibrosis Transmembrane Conductance Regulator, Reviews, Disease, Bioinformatics, Cystic fibrosis, 03 medical and health sciences, 0302 clinical medicine, Genome editing, Genetics, medicine, Humans, CRISPR, Molecular Biology, Gene, 030304 developmental biology, Gene Editing, 0303 health sciences, Pulmonary Surfactant-Associated Protein A, biology, business.industry, Genetic Therapy, Dependovirus, medicine.disease, biology.organism_classification, alpha 1-Antitrypsin, 030220 oncology & carcinogenesis, Lentivirus, Molecular Medicine, business, Ex vivo
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ddee8379ab30d0b327108315608f7407
https://doi.org/10.1089/hum.2020.166 -
6
المؤلفون: Christian Mueller, Chun-Qing Song, Lingling Cai, Tingting Jiang, Hao Yin, Dan Wang, Wen Xue, Kevin W O'Connor, Terence R. Flotte, Xiangrui Li, Guangping Gao, Qiushi Tang, Zhiping Weng
المصدر: Human Gene Therapy. 29:853-860
مصطلحات موضوعية: 0301 basic medicine, congenital, hereditary, and neonatal diseases and abnormalities, Genetic Vectors, Mice, Transgenic, Biology, medicine.disease_cause, Deep sequencing, Mice, 03 medical and health sciences, Liver disease, Genome editing, alpha 1-Antitrypsin Deficiency, Genetics, medicine, Animals, Humans, CRISPR, Guide RNA, Molecular Biology, Gene, Research Articles, Gene Editing, Mutation, Cas9, Genetic Therapy, Dependovirus, medicine.disease, Disease Models, Animal, 030104 developmental biology, alpha 1-Antitrypsin, Molecular Medicine, CRISPR-Cas Systems
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a2425226da5896235635ac137cbc09bd
https://doi.org/10.1089/hum.2017.225 -
7
المؤلفون: Charles F Albright, Haiyan Jiang, Minerva E Sanchez, Eugenio Marco, Jeffrey Teckman, Keith Blomenkamp, Shen Shen, Erik M Corcoran, Clifford Yudkoff, David Bumcrot
المصدر: Human Gene Therapy. 29:861-873
مصطلحات موضوعية: 0301 basic medicine, Genetically modified mouse, congenital, hereditary, and neonatal diseases and abnormalities, Cirrhosis, Genetic enhancement, Genetic Vectors, Mice, Transgenic, Biology, medicine.disease_cause, Mice, 03 medical and health sciences, Exon, Liver disease, alpha 1-Antitrypsin Deficiency, Genetics, medicine, Animals, Humans, Molecular Biology, Gene, Alleles, Gene Editing, Mutation, Alpha 1-antitrypsin deficiency, Homozygote, Genetic Therapy, Dependovirus, medicine.disease, Molecular biology, Disease Models, Animal, 030104 developmental biology, Liver, alpha 1-Antitrypsin, Hepatocytes, Molecular Medicine, CRISPR-Cas Systems
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9fbb6898f93b170875e8aa11288389ad
https://doi.org/10.1089/hum.2017.227 -
8
المؤلفون: Ronald G. Crystal
المصدر: Human Gene Therapy. 31:273-282
مصطلحات موضوعية: Career Choice, Cystic Fibrosis, business.industry, Genetic enhancement, Genetic Therapy, Neuronal Ceroid-Lipofuscinoses, alpha 1-Antitrypsin Deficiency, Genetics, Cancer research, Humans, Molecular Medicine, Medicine, business, Molecular Biology
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cb697f09134fcfbb846a264c52b4a36e
https://doi.org/10.1089/hum.2020.29112.rgc -
9
المؤلفون: Martin Rüdiger, Chun-wa Chung, Jonathan P. Hutchinson, Christopher C. Arico-Muendel, Svetlana L. Belyanskaya, Allison Olszewski, Nerina Dodic, Duncan S. Holmes, Anthony Dossang, Andrew C. Pearce, Alistair M. Jagger, Steve Wilson, Adriana Ordóñez, David A. Lomas, Toral Jakhria, Iain Uings, Hitesh Dave, Zhengrong Zhu, Stefan J. Marciniak, Alexis Denis, Lionel Trottet, Kathrine J. Smith, Murray J. B. Brown, Imran Haq, James A. Irving, Steve Skinner, Margaret Neu, Diana Klimaszewska, Peter Eddershaw, Riccardo Ronzoni, James E. Rowedder, Andrew Brewster, John Liddle, Emilie Jigorel, Jeffrey A. Messer, Ken Lind, Rebecca Terry
المساهمون: Lomas, David A [0000-0003-2339-6979], Irving, James A [0000-0003-3204-6356], Ronzoni, Riccardo [0000-0002-3981-8104], Pearce, Andrew C [0000-0002-4698-037X], Apollo - University of Cambridge Repository
المصدر: EMBO Molecular Medicine
EMBO Molecular Medicine, Vol 13, Iss 3, Pp n/a-n/a (2021)مصطلحات موضوعية: 0301 basic medicine, Genetically modified mouse, Medicine (General), Mutant, α1-antitrypsin deficiency, QH426-470, Endoplasmic Reticulum, medicine.disease_cause, Article, Mice, 03 medical and health sciences, R5-920, 0302 clinical medicine, alpha 1-Antitrypsin Deficiency, Chemical Biology, Genetics, small molecule corrector, medicine, Animals, Secretion, protein misfolding, Mutation, Chemistry, Endoplasmic reticulum, Articles, Molecular biology, Small molecule, In vitro, emphysema, 030104 developmental biology, alpha 1-Antitrypsin, Hepatocytes, Molecular Medicine, Genetics, Gene Therapy & Genetic Disease, liver disease, 030217 neurology & neurosurgery, Intracellular, α1‐antitrypsin deficiency
وصف الملف: application/pdf
-
10
المؤلفون: Barbara A. Osborne, Ulf Geumann, Sudarvili Shanthalingam, Lisa M. Minter, Stefanie Kälin, Terese Magnusson, Daniela Hirsch, Gregory N. Tew, Felix Hermann, Sabine Geiger, Christine Günther, Emrah Ilker Ozay, Marina K. Hereth
المصدر: Mol Ther
مصطلحات موضوعية: Regulatory T cell, medicine.medical_treatment, Genetic Vectors, Gene Expression, Graft vs Host Disease, Alpha (ethology), Hematopoietic stem cell transplantation, Disease, Lymphocyte Activation, Mesenchymal Stem Cell Transplantation, T-Lymphocytes, Regulatory, Mice, 03 medical and health sciences, 0302 clinical medicine, Gene Order, Drug Discovery, Genetics, medicine, Animals, Transplantation, Homologous, Protease inhibitor (pharmacology), Molecular Biology, 030304 developmental biology, Pharmacology, 0303 health sciences, business.industry, Macrophages, Mesenchymal stem cell, Mesenchymal Stem Cells, Dependovirus, M2 Macrophage, medicine.disease, Chemotaxis, Leukocyte, Disease Models, Animal, Treatment Outcome, medicine.anatomical_structure, Graft-versus-host disease, Organ Specificity, alpha 1-Antitrypsin, 030220 oncology & carcinogenesis, Cancer research, Cytokines, Heterografts, Molecular Medicine, Original Article, Inflammation Mediators, business
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b9afab4ffc97a3e93f1d6ac4414a3326
https://doi.org/10.1016/j.ymthe.2019.05.007
إعادة تعيين المنقحات
عرض المنقحات (%%عد%%)