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1دورية أكاديمية
المؤلفون: Yildirim Dogan, Cecilia N. Barese, Jeffrey W. Schindler, John K. Yoon, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Christine Oborski, Tim Maiwald, Diana L. Clarke, Axel Schambach, Richard Pfeifer, Claudia Harper, Chris Mason, Niek P. van Til
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 464-487 (2022)
مصطلحات موضوعية: hematopoietic stem and progenitor cells, lentiviral vector, Pompe disease, tag technology, glycosylation-independent lysosomal targeting, Genetics, QH426-470, Cytology, QH573-671
وصف الملف: electronic resource
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2دورية أكاديمية
المؤلفون: Christian Brendel, Olivier Negre, Michael Rothe, Swaroopa Guda, Geoff Parsons, Chad Harris, Meaghan McGuinness, Daniela Abriss, Alla Tsytsykova, Denise Klatt, Martin Bentler, Danilo Pellin, Lauryn Christiansen, Axel Schambach, John Manis, Helene Trebeden-Negre, Melissa Bonner, Erica Esrick, Gabor Veres, Myriam Armant, David A. Williams
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 589-600 (2020)
مصطلحات موضوعية: Sickle cell disease, HbS, hemoglobinopathies, gene therapy, lentiviral vector, BCL11A, Genetics, QH426-470, Cytology, QH573-671
وصف الملف: electronic resource
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3دورية أكاديمية
المؤلفون: Hee Ho Park, Robinson Triboulet, Martin Bentler, Swaroopa Guda, Peng Du, Haiming Xu, Richard I. Gregory, Christian Brendel, David A. Williams
المصدر: Molecular Therapy: Nucleic Acids, Vol 12, Iss , Pp 591-599 (2018)
مصطلحات موضوعية: Therapeutics. Pharmacology, RM1-950
وصف الملف: electronic resource
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المؤلفون: Yildirim Dogan, Cecilia N. Barese, Jeffrey W. Schindler, John K. Yoon, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Christine Oborski, Diana L. Clarke, Axel Schambach, Richard Pfeifer, Claudia Harper, Chris Mason, Niek P. van Til
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::89f466bcec75e33a143d065b6b9e12b3
https://doi.org/10.1101/2021.12.28.474352 -
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المؤلفون: Olivier Negre, Swaroopa Guda, Christian Brendel, Chad E. Harris, Martin Bentler, Myriam Armant, Melissa Bonner, Erica B. Esrick, John P. Manis, Helene Trebeden-Negre, Axel Schambach, Alla V. Tsytsykova, Danilo Pellin, Michael Rothe, Lauryn Christiansen, Denise Klatt, David A. Williams, Meaghan McGuinness, Daniela Abriss, Geoff Parsons, Gabor Istvan Veres
المصدر: Molecular Therapy: Methods & Clinical Development, Vol 17, Iss, Pp 589-600 (2020)
Molecular Therapy. Methods & Clinical Developmentمصطلحات موضوعية: 0301 basic medicine, fetal hemoglobin, lcsh:QH426-470, Genetic enhancement, Cell, CD34, Biology, Article, Viral vector, Small hairpin RNA, 03 medical and health sciences, 0302 clinical medicine, RNA interference, BCL11A, Fetal hemoglobin, Genetics, medicine, shRNAmiR, lcsh:QH573-671, hemoglobinopathies, Molecular Biology, Gene knockdown, lcsh:Cytology, Sickle cell disease, lentiviral vector, gene therapy, lcsh:Genetics, 030104 developmental biology, medicine.anatomical_structure, HbS, 030220 oncology & carcinogenesis, hemoglobin switch, Cancer research, Molecular Medicine
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المؤلفون: Niek P. van Til, Jeffrey W. Schindler, John K. Yoon, Yildirim Dogan, Cecilia Barese, Zeenath Unnisa, Swaroopa Guda, Mary E. Jacobs, Daniella Pizzurro, Mirjam Trame, Tim Maiwald, Christine Oborski, Michael Rothe, Axel Schambach, Claudia Harper, Richard Pfeifer, Chris Mason
المصدر: Molecular Genetics and Metabolism. 135:S122-S123
مصطلحات موضوعية: Endocrinology, Endocrinology, Diabetes and Metabolism, Genetics, Molecular Biology, Biochemistry
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المؤلفون: Abhishek Chiyyeadu, Maurine Braun, Claudia Harper, Christopher E. Mason, Christine Oborski, Daniella Pizzurro, Axel Schambach, Niek P. van Til, Bianling Liu, John K. Yoon, Yildirim Dogan, Richard Pfeifer, Swaroopa Guda, Cecilia Barese, Zeenath Unnisa, Mary Jacobs, Rena Schindler
المصدر: Molecular Genetics and Metabolism. 132:S107
مصطلحات موضوعية: business.industry, Endocrinology, Diabetes and Metabolism, Genetic enhancement, Hematopoietic stem cell, Biochemistry, Term (time), Endocrinology, medicine.anatomical_structure, Genetics, Cancer research, Medicine, business, Molecular Biology, Neuromuscular Manifestations
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المؤلفون: Swaroopa Guda, Michael D. Milsom, Peng Du, Christian Brendel, David E. Root, Sophia C. Kamran, Matthew C. Canver, Andrew Grimson, Richard I. Gregory, Raffaele Renella, Daniel E. Bauer, Jennifer K. Grenier, Stuart H. Orkin, Helen de Boer, David A. Williams, James E. Thornton
المصدر: Molecular Therapy. 23(9):1465-1474
مصطلحات موضوعية: Genetic Vectors, Gene Expression, Biology, Cell Line, Small hairpin RNA, 03 medical and health sciences, Mice, 0302 clinical medicine, Erythroid Cells, RNA interference, Transduction, Genetic, microRNA, Gene expression, Gene Order, Drug Discovery, Gene Knockdown Techniques, Genetics, Gene silencing, Animals, Humans, Cell Lineage, gamma-Globins, Gene Silencing, RNA, Small Interfering, Promoter Regions, Genetic, Molecular Biology, Fetal Hemoglobin, 030304 developmental biology, Pharmacology, 0303 health sciences, Gene knockdown, Base Sequence, Nuclear Proteins, Molecular biology, Cell biology, Repressor Proteins, MicroRNAs, Retroviridae, 030220 oncology & carcinogenesis, Hemoglobin F, Nucleic Acid Conformation, Molecular Medicine, RNA Interference, Original Article, Carrier Proteins
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المؤلفون: Martin Bentler, Christian Brendel, Peng Du, Haiming Xu, Richard I. Gregory, Hee Ho Park, Robinson Triboulet, David A. Williams, Swaroopa Guda
المصدر: Molecular Therapy. Nucleic Acids
Molecular Therapy: Nucleic Acids, Vol 12, Iss, Pp 591-599 (2018)مصطلحات موضوعية: 0301 basic medicine, Messenger RNA, lcsh:RM1-950, Biology, Article, 3. Good health, Cell biology, Microprocessor complex, Small hairpin RNA, 03 medical and health sciences, lcsh:Therapeutics. Pharmacology, 030104 developmental biology, DROSHA microprocessor, RNA interference, RNAi, Drug Discovery, Gene expression, microRNA, producer cell line, Molecular Medicine, Northern blot, recombinant viral vector titer, CRISPR/Cas9, Drosha
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المؤلفون: Matthew C. Canver, Jonathan Fogel, Swaroopa Guda, Christian Brendel, Daniel E. Bauer, Richard I. Gregory, Stuart H. Orkin, David A. Williams, Denise Klatt, Matthew M. Heeney, Young-Jo Kim, Raffaele Renella, Michael D. Milsom
مصطلحات موضوعية: 0301 basic medicine, Hemolytic anemia, Reticulocytosis, medicine.medical_treatment, Graft vs Host Disease, Antigens, CD34, Hematopoietic stem cell transplantation, Anemia, Sickle Cell, Mice, SCID, 03 medical and health sciences, 0302 clinical medicine, Mice, Inbred NOD, hemic and lymphatic diseases, Fetal hemoglobin, medicine, Animals, Humans, Cell Lineage, RNA, Small Interfering, Cells, Cultured, Genetics, Gene knockdown, business.industry, Hematopoietic Stem Cell Transplantation, Nuclear Proteins, General Medicine, Genetic Therapy, medicine.disease, Hematopoietic Stem Cells, Hematopoiesis, Transplantation, Mice, Inbred C57BL, Repressor Proteins, Haematopoiesis, 030104 developmental biology, Phenotype, 030220 oncology & carcinogenesis, Gene Knockdown Techniques, Cancer research, medicine.symptom, Stem cell, business, Carrier Proteins, Research Article
URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::37dd4658e09edd89ffcf9c6744cd7a8c
https://europepmc.org/articles/PMC5096824/