المؤلفون: Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams

المصدر: Blood. 140:10665-10667

مصطلحات موضوعية: Immunology, Cell Biology, Hematology, Biochemistry

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::e828f889a6fcfac4de9b7fcb45370915
https://doi.org/10.1182/blood-2022-169089

المؤلفون: Donald B. Kohn, Claire Booth, Kit L. Shaw, Jinhua Xu-Bayford, Elizabeth Garabedian, Valentina Trevisan, Denise A. Carbonaro-Sarracino, Kajal Soni, Dayna Terrazas, Katie Snell, Alan Ikeda, Diego Leon-Rico, Theodore B. Moore, Karen F. Buckland, Ami J. Shah, Kimberly C. Gilmour, Satiro De Oliveira, Christine Rivat, Gay M. Crooks, Natalia Izotova, John Tse, Stuart Adams, Sally Shupien, Hilory Ricketts, Alejandra Davila, Chilenwa Uzowuru, Amalia Icreverzi, Provaboti Barman, Beatriz Campo Fernandez, Roger P. Hollis, Maritess Coronel, Allen Yu, Krista M. Chun, Christian E. Casas, Ruixue Zhang, Serena Arduini, Frances Lynn, Mahesh Kudari, Andrea Spezzi, Marco Zahn, Rene Heimke, Ivan Labik, Roberta Parrott, Rebecca H. Buckley, Lilith Reeves, Kenneth Cornetta, Robert Sokolic, Michael Hershfield, Manfred Schmidt, Fabio Candotti, Harry L. Malech, Adrian J. Thrasher, H. Bobby Gaspar

المصدر: N Engl J Med
The New England journal of medicine, vol 384, iss 21

مصطلحات موضوعية: Adenosine Deaminase, Genetic enhancement, 030204 cardiovascular system & hematology, Regenerative Medicine, Medical and Health Sciences, 0302 clinical medicine, Adenosine deaminase, Stem Cell Research - Nonembryonic - Human, Agammaglobulinemia, immune system diseases, Medicine, Prospective Studies, 030212 general & internal medicine, Child, 6.2 Cellular and gene therapies, biology, Hematopoietic Stem Cell Transplantation, hemic and immune systems, Gene Therapy, General Medicine, Progression-Free Survival, Child, Preschool, Development of treatments and therapeutic interventions, Autologous, Biotechnology, congenital, hereditary, and neonatal diseases and abnormalities, Adolescent, Genetic Vectors, Transplantation, Autologous, Article, 03 medical and health sciences, Clinical Research, General & Internal Medicine, Genetics, Humans, Lymphocyte Count, Preschool, Transplantation, Severe combined immunodeficiency, 5.2 Cellular and gene therapies, business.industry, Extramural, Lentivirus, Evaluation of treatments and therapeutic interventions, Infant, nutritional and metabolic diseases, Genetic Therapy, Stem Cell Research, medicine.disease, Adenosine deaminase deficiency, enzymes and coenzymes (carbohydrates), Good Health and Well Being, Primary immunodeficiency, Cancer research, biology.protein, Severe Combined Immunodeficiency, business, Ex vivo

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b51d435955d4b74762af96e7db7ff4e2
https://doi.org/10.1056/nejmoa2027675

المؤلفون: Kit L. Shaw, Kathryn L. Bradford, Maja Krajinovic, Siyu Liu, Xiaoyan Wang, Elizabeth Garabedian, John Tse, Donald B. Kohn, Marc Ansari, Fabio Candotti, H. Bobby Gaspar

المصدر: Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, vol 26, iss 10
Biology of blood and marrow transplantation, Vol. 26, No 10 (2020) pp. 1819-1827

مصطلحات موضوعية: Oncology, Transplantation Conditioning, Adenosine Deaminase, Genetic enhancement, Severe Combined Immunodeficiency / therapy, Regenerative Medicine, Adenosine Deaminase / genetics, Clinical trials, 0302 clinical medicine, Agammaglobulinemia, Stem Cell Research - Nonembryonic - Human, Child, Body surface area, ddc:618, medicine.diagnostic_test, Hematopoietic Stem Cell Transplantation, Hematology, medicine.anatomical_structure, 030220 oncology & carcinogenesis, Stem Cell Research - Nonembryonic - Non-Human, Biotechnology, medicine.drug, medicine.medical_specialty, Clinical Trials and Supportive Activities, Clinical Sciences, Immunology, SCID, 03 medical and health sciences, Gene therapy, Pharmacokinetics, Clinical Research, Internal medicine, Genetics, medicine, Humans, Dosing, Busulfan, Transplantation, Severe combined immunodeficiency, business.industry, Infant, Genetic Therapy, Stem Cell Research, medicine.disease, Severe Combined Immunodeficiency / genetics, Therapeutic drug monitoring, Severe Combined Immunodeficiency, Bone marrow, business, 030215 immunology

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::77dc50696f7bea24f30dd28cc2d327b1
https://doi.org/10.1016/j.bbmt.2020.07.004

المؤلفون: Glenn J. Hanna, Kimberly Coleman, Grace Birch, Robert A. Redd, Alejandro Alonso, Samantha Bednarz, Heather Daley, Diego E. Hernandez Rodriguez, Kit L. Shaw, Robert I. Haddad, Ravindra Uppaluri, Jerome Ritz, Sarah Nikiforow, Robert J. Soiffer, Rizwan Romee

المصدر: Cancer Research. 82:CT540-CT540

مصطلحات موضوعية: Cancer Research, Oncology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::51bd1e157fd79716f490bcbcfd4a65aa
https://doi.org/10.1158/1538-7445.am2022-ct540

المؤلفون: Sally Shupien, Aoife M. Roche, Dayna Terrazas, Augustine Fernandes, Satiro N. De Oliveira, Eline T. Luning Prak, Jack Mottahedeh, Omar Habib, Xiaoyan Wang, Shantan Reddy, Wenzhao Meng, Theodore B. Moore, Pascha Hokama, Michael S. Hershfield, Suk See De Ravin, Connie Jackson, Beatriz Campo Fernandez, Andrej Vitomirov, Kenneth Cornetta, Denise A. Carbonaro-Sarracino, Kit L. Shaw, Robert A. Sokolic, Elizabeth Garabedian, Harry L. Malech, Barbara C. Engel, Alan K. Ikeda, Aaron M. Rosenfeld, Gregory M. Podsakoff, Bryanna Reinhardt, Donald B. Kohn, Fabio Candotti, Frederic D. Bushman, John K. Everett, Alejandra Davila, Suparna Mishra, Roger P. Hollis

المصدر: Blood

مصطلحات موضوعية: Adolescent, Adenosine Deaminase, medicine.medical_treatment, Genetic enhancement, Immunology, Hematopoietic stem cell transplantation, Biochemistry, Transplantation, Autologous, Immune system, Adenosine deaminase, Agammaglobulinemia, medicine, Humans, Child, Severe combined immunodeficiency, biology, business.industry, Hematopoietic Stem Cell Transplantation, Infant, Cell Biology, Hematology, Enzyme replacement therapy, Genetic Therapy, Gene Therapy, medicine.disease, Treatment Outcome, Child, Preschool, biology.protein, Severe Combined Immunodeficiency, Antibody, business, Busulfan, medicine.drug, Follow-Up Studies

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6cb34b0c6a6fdfaf418692ef5a41e734
https://pubmed.ncbi.nlm.nih.gov/34647983

المؤلفون: Kit L. Shaw, Irene Scarfò, Lu Huang, Thomas R. Spitzer, Areej El-Jawahri, Steven L. McAfee, Matthew J. Frigault, Frederic I. Preffer, Kathleen Gallagher, Heather Daley, Nora Horick, Myriam Armant, Marcela V. Maus, Jerome Ritz, Daniella Cook, Keagan S Casey, Sarah Nikiforow, Yi-Bin Chen, Marc Wehrli

المصدر: Blood. 138:653-653

مصطلحات موضوعية: Refractory, business.industry, Phase (matter), Immunology, Cancer research, Medicine, In patient, Cell Biology, Hematology, CD37, Car t cells, business, Biochemistry

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::974b7f12e9dfe9d6d3ea966dc191309f
https://doi.org/10.1182/blood-2021-146236

المؤلفون: Rao Prabhala, Diego Hernandez Rodriguez, Kenneth C. Anderson, Marc Credi, Lawrence Rispoli, Omar Nadeem, Jacob P. Laubach, Sarah Nikiforow, Kit L. Shaw, Nikhil C. Munshi, Yohei Arihara, Soudeh Ansari, Jerome Ritz, Alejandro Alonso, Serena De Vita, Shuntaro Ikegawa, David Pearson, Clifton C. Mo, Jennifer Marie Mataraza, Anniesha Hack, Adam S. Sperling, Dexiu Bu, Heather Daley, Louise Treanor, David Quinn

المصدر: Blood. 138:3864-3864

مصطلحات موضوعية: business.industry, Immunology, Charge (physics), Cell Biology, Hematology, medicine.disease, Biochemistry, Phase i study, Relapsed refractory, Cancer research, Medicine, CAR T-cell therapy, business, Multiple myeloma

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::7d7b987e0d01e2809cdf444935b3bb41
https://doi.org/10.1182/blood-2021-146646

المؤلفون: Matteo Pellegrini, Denise A. Carbonaro-Sarracino, Robert A. Sokolic, Aaron R. Cooper, Donald B. Kohn, Alejandra Davila, Fabio Candotti, Kit L. Shaw, Georgia R. Lill

المصدر: Blood. 129:2624-2635

مصطلحات موضوعية: 0301 basic medicine, LMO2, MECOM, Adenosine Deaminase, T-Lymphocytes, Genetic enhancement, Genetic Vectors, Immunology, Viremia, Biology, Biochemistry, Virus, 03 medical and health sciences, 0302 clinical medicine, Agammaglobulinemia, Proto-Oncogene Proteins, Proto-Oncogenes, medicine, Humans, Child, Antineoplastic Agents, Alkylating, Busulfan, Adaptor Proteins, Signal Transducing, Severe combined immunodeficiency, Genetic Therapy, Gene Therapy, Cell Biology, Hematology, respiratory system, LIM Domain Proteins, medicine.disease, MDS1 and EVI1 Complex Locus Protein, Adenosine deaminase deficiency, DNA-Binding Proteins, Leukemia, 030104 developmental biology, 030220 oncology & carcinogenesis, Severe Combined Immunodeficiency, Gammaretrovirus, human activities, Transcription Factors

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::17ddaa21f44c4bd12abb16ef09eac6f7
https://doi.org/10.1182/blood-2016-12-756734

المؤلفون: Sally Shupien, Linda M. Muul, Fabio Candotti, Berkley Brown, Pei Yu Fu, Rob Sokolic, Barbara Nowicki, Otto O. Yang, Aaron R. Cooper, Andrew M. Scharenberg, Christopher Silvin, David W. Gjertson, Alejandra Davila, Kit L. Shaw, Dayna Terrazas, Donald B. Kohn, Alan Ikeda, Beatriz Campo Fernandez, Radha G. Rishi, Neena Kapoor, Xiaoyan Wang, Denise Carbonaro, Suparna Mishra, Arumugam Balamurugan, G. Jayashree Jagadeesh, Satiro N. De Oliveira, Yeong Choi, Kenneth Cornetta, Suzanne Skoda-Smith, Michael S. Hershfield, Roshini S. Abraham, Sabine Geiger, David Buchbinder, Barbara C. Engel, Provaboti Barman, Theodore B. Moore, Kathey Mohan, E. Monika Smogorzewska, Elizabeth Garabedian, Allen Yu, Ken Purdy, John Tse, Greg M. Podsakoff, Stacie M. Anderson

المصدر: Journal of Clinical Investigation. 127:1689-1699

مصطلحات موضوعية: Male, 0301 basic medicine, Oncology, congenital, hereditary, and neonatal diseases and abnormalities, medicine.medical_specialty, Adolescent, Adenosine Deaminase, Genetic enhancement, medicine.medical_treatment, Genetic Vectors, CD34, Hematopoietic stem cell transplantation, Gene Expression Regulation, Enzymologic, 03 medical and health sciences, Agammaglobulinemia, Transduction, Genetic, immune system diseases, hemic and lymphatic diseases, Internal medicine, medicine, Humans, Progenitor cell, Autografts, Child, business.industry, Hematopoietic Stem Cell Transplantation, Infant, nutritional and metabolic diseases, Genetic Therapy, General Medicine, medicine.disease, 3. Good health, Adenosine deaminase deficiency, Transplantation, Retroviridae, surgical procedures, operative, 030104 developmental biology, Child, Preschool, Female, Severe Combined Immunodeficiency, Clinical Medicine, Stem cell, business, Busulfan, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c7495591a0b8eb59cc66fe20e2a54576
https://doi.org/10.1172/jci90367

المؤلفون: Natalia Izotova, Geraldine Honnet, Myriam Armant, Luca Biasco, Giorgia Santilli, Christopher A. Bauser, Katie Snell, Suk See De Ravin, Karen F. Buckland, Emma C. Morris, Morna J. Dorsey, Peter E. Newburger, Jinan Darwish, Christine Rivat, Diego Leon-Rico, David A. Williams, Adrian J. Thrasher, Kit L. Shaw, Kimberly Gilmour, Sung-Yun Pai, Leo D. Wang, Donald B. Kohn, Tobias Paprotka, John R. Gregg, Claire Booth, Harry L. Malech, Uimook Choi, Caroline Y. Kuo, Elizabeth M. Kang, Manuel Grez, Jinhua Xu-Bayford Dip, Douglas B. Kuhns, John K. Everett, H. Bobby Gaspar, Frederic D. Bushman, Hayley Raymond, Anne Galy, Dayna Terrazas

المساهمون: Institut des Neurosciences de Montpellier - Déficits sensoriels et moteurs (INM), Université de Montpellier (UM)-Institut National de la Santé et de la Recherche Médicale (INSERM), 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, Institut des Neurosciences de Montpellier (INM), Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Montpellier (UM)

المصدر: Nature medicine, vol 26, iss 2
Nature Medicine
Nature Medicine, Nature Publishing Group, 2020, ⟨10.1038/s41591-019-0735-5⟩
Nature Medicine, 2020, ⟨10.1038/s41591-019-0735-5⟩
Nat Med

مصطلحات موضوعية: 0301 basic medicine, Male, Transplantation Conditioning, Neutrophils, Genetic enhancement, [SDV]Life Sciences [q-bio], CD34, Antigens, CD34, Comorbidity, Granulomatous Disease, Chronic, Regenerative Medicine, Medical and Health Sciences, 0302 clinical medicine, Chronic granulomatous disease, Stem Cell Research - Nonembryonic - Human, Genes, Regulator, Medicine, Antibiotic prophylaxis, Chronic, Promoter Regions, Genetic, Child, Hematopoietic stem cell, General Medicine, Hematology, Gene Therapy, 3. Good health, Haematopoiesis, medicine.anatomical_structure, Treatment Outcome, Infectious Diseases, Child, Preschool, 030220 oncology & carcinogenesis, Granulomatous Disease, Patient Safety, Development of treatments and therapeutic interventions, Infection, Human, Adolescent, Genetic Vectors, Clinical Trials and Supportive Activities, Immunology, Article, General Biochemistry, Genetics and Molecular Biology, Chromosomes, Promoter Regions, 03 medical and health sciences, Young Adult, Genetic, Immunity, Clinical Research, Genetics, Humans, Gene Silencing, Progenitor cell, Antigens, Preschool, Chromosomes, Human, X, 5.2 Cellular and gene therapies, business.industry, Inflammatory and immune system, Lentivirus, Regulator, NADPH Oxidases, Genetic Therapy, medicine.disease, Hematopoietic Stem Cells, Stem Cell Research, Net4CGD consortium, United States, United Kingdom, 030104 developmental biology, Genes, business

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7cfc100375d6c5f54fdce1801ee6c80b
https://escholarship.org/uc/item/7b61w03m

المؤلفون: Kit L. Shaw, Dirk Lindemann, Mark J. Mulligan, Paul A

المساهمون: The Pennsylvania State University CiteSeerX Archives

المصدر: http://jvi.asm.org/content/77/4/2338.full.pdf.

وصف الملف: application/pdf

Relation: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.535.6025; http://jvi.asm.org/content/77/4/2338.full.pdf

الاتاحة: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.535.6025
http://jvi.asm.org/content/77/4/2338.full.pdf

المؤلفون: P A Goepfert, K L Shaw, G D Ritter, M J Mulligan, Endoplasmic Reticulum, Paul A. Goepfert, Kit L. Shaw, G. Douglas Ritter, Mark J. Mulligan

المساهمون: The Pennsylvania State University CiteSeerX Archives

المصدر: http://jvi.asm.org/content/71/1/778.full.pdf.

وصف الملف: application/pdf

Relation: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.615.1824; http://jvi.asm.org/content/71/1/778.full.pdf

الاتاحة: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.615.1824
http://jvi.asm.org/content/71/1/778.full.pdf

المؤلفون: Donald B. Kohn, Kit L. Shaw, Elizabeth Garabedian, Denise Ann Carbonaro-Sarracino, Theodore B. Moore, Satiro N. De Oliveira, Gay M. Crooks, John Tse, Sally Shupien, Dayna Terrazas, Alejandra Davila, Amalia Icreverzi, Allen Yu, Krista M. Chun, Christian E. Casas, Provaboti Barman, Maritess Coronel, Beatriz Campo Fernandez, Ruixue Zhang, Roger P. Hollis, Chilenwa Uzowuru, Hilory Ricketts, Jinhua Xu Bayford, Valentina Trevisan, Serena Arduini, Frances Lynn, Mahesh Kudari, Andrea Spezzi, Lilith Reeves, Kenneth Cornetta, Robert A. Sokolic, Roberta Parrott, Rebecca Buckley, Claire Booth, Fabio Candotti, Harry L. Malech, Adrian J. Thrasher, H. Bobby Gaspar

المصدر: Blood. 134:3345-3345

مصطلحات موضوعية: medicine.medical_specialty, business.industry, medicine.medical_treatment, Immunology, Cell Biology, Hematology, Hematopoietic stem cell transplantation, Gene mutation, medicine.disease, Biochemistry, Adenosine deaminase deficiency, Clinical trial, Graft-versus-host disease, Internal medicine, Cohort, medicine, business, Busulfan, Immunodeficiency, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::22a9f018d10bc165f3940826b9e244c9
https://doi.org/10.1182/blood-2019-123432

المؤلفون: Fabio Candotti, Kit L. Shaw, Linda Muul, Denise Carbonaro, Robert Sokolic, Christopher Choi, Shepherd H. Schurman, Elizabeth Garabedian, Chimene Kesserwan, G. Jayashree Jagadeesh, Pei-Yu Fu, Eric Gschweng, Aaron Cooper, John F. Tisdale, Kenneth I. Weinberg, Gay M. Crooks, Neena Kapoor, Ami Shah, Hisham Abdel-Azim, Xiao-Jin Yu, Monika Smogorzewska, Alan S. Wayne, Howard M. Rosenblatt, Carla M. Davis, Celine Hanson, Radha G. Rishi, Xiaoyan Wang, David Gjertson, Otto O. Yang, Arumugam Balamurugan, Gerhard Bauer, Joanna A. Ireland, Barbara C. Engel, Gregory M. Podsakoff, Michael S. Hershfield, R. Michael Blaese, Robertson Parkman, Donald B. Kohn

المصدر: Blood. 120:3635-3646

مصطلحات موضوعية: Male, Transplantation Conditioning, Adenosine Deaminase, medicine.medical_treatment, Genetic enhancement, Genetic Vectors, Immunology, Plenary Paper, Hematopoietic stem cell transplantation, Biochemistry, Adenosine deaminase, Agammaglobulinemia, medicine, Animals, Humans, Bone Marrow Transplantation, Severe combined immunodeficiency, biology, business.industry, Hematopoietic Stem Cell Transplantation, nutritional and metabolic diseases, Genetic Therapy, Cell Biology, Hematology, Enzyme replacement therapy, medicine.disease, medicine.anatomical_structure, biology.protein, Female, Severe Combined Immunodeficiency, Bone marrow, business, Busulfan, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ee6368c410c1271964561f3a41d89826
https://doi.org/10.1182/blood-2012-02-400937

المؤلفون: Dianne C. Skelton, Shundi Ge, Kit L. Shaw, Eszter Pais, Crooks Gm, Donald B. Kohn, Roger P. Hollis, Cinnamon L Hardee

المصدر: Gene therapy
Gene therapy, vol 16, iss 8

مصطلحات موضوعية: Transcriptional Activation, GAL4/UAS system, Therapeutic gene modulation, lineage-specificity, gene amplification, Genetic Vectors, Green Fluorescent Proteins, insulin promoter, Gene Expression, Biology, Medical and Health Sciences, Article, Cell Line, Promoter Regions, Islets of Langerhans, Mice, 03 medical and health sciences, 0302 clinical medicine, Genetic, Insulin-Secreting Cells, Gene expression, Genetics, Animals, Humans, Insulin, Promoter Regions, Genetic, Molecular Biology, 030304 developmental biology, Regulator gene, 0303 health sciences, Reporter gene, Expression vector, Activator (genetics), lentiviral vector, Lentivirus, Gene Transfer Techniques, Biological Sciences, Molecular biology, Phosphoglycerate Kinase, Organ Specificity, 030220 oncology & carcinogenesis, Molecular Medicine, Heterologous expression, Biotechnology

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::69d6c588d507ea3c8b5d58bb2325a50b
https://doi.org/10.1038/gt.2009.49

المؤلفون: Fabien Touzot, Gregory Hopkins, Sung-Yun Pai, Salima Hacein-Bey-Abina, Frances Male, Myriam Armant, Despina Moshous, Luigi D. Notarangelo, David A. Williams, Jerome Ritz, Capucine Picard, Helen de Boer, Annick Lim, Karen F. Buckland, Dongjing Guo, Axel Schambach, Laure Caccavelli, Johannes C.M. Van der Loo, Satiro N. De Oliveira, Leslie Lehmann, Punam Malik, Alexandra H. Filipovich, Johanna Blondeau, M. Angélica Marinovic, Stéphane Blanche, Kit L. Shaw, Charles C. Berry, Alla V. Tsytsykova, Karen S. Fernandez, Alain Fischer, Eric A. Sherman, Anne Marie McNicol, Leslie E. Silberstein, Guilhem Cros, Adrian J. Thrasher, Bénédicte Neven, H. Bobby Gaspar, Frederic D. Bushman, Matias Oleastro, Nirav Malani, Marina Cavazzana, Donald B. Kohn, Jack J. Bleesing, Christine Rivat, Wendy B. London, Chad E. Harris, Christopher Baum, Emmanuelle Six, Jinhua Xu-Bayford

المصدر: Hacein-Bey-Abina, S; Pai, SY; Gaspar, HB; Armant, M; Berry, CC; Blanche, S; et al.(2014). A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. New England Journal of Medicine, 371(15), 1407-1417. doi: 10.1056/NEJMoa1404588. UCLA: Retrieved from: http://www.escholarship.org/uc/item/2bn9c1nt

مصطلحات موضوعية: Male, DNA, Complementary, MECOM, T-Lymphocytes, Genetic Vectors, Gene Expression, Antigens, CD34, X-Linked Combined Immunodeficiency Diseases, Article, Mice, Antigen, Transduction, Genetic, Medicine, Animals, Humans, X-linked severe combined immunodeficiency, Gene Silencing, Transgenes, Gammaretrovirus, Severe combined immunodeficiency, biology, business.industry, Infant, General Medicine, Genetic Therapy, medicine.disease, biology.organism_classification, Virology, Transplantation, Leukemia, Immunology, Mutation, business, Interleukin Receptor Common gamma Subunit

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::96c2567d602f6e62c57cf31b5a1d09c6
https://europepmc.org/articles/PMC4274995/

المؤلفون: Kit L. Shaw, Anju Bansal, Bradley H. Edwards, Mark J. Mulligan, George Wang, Paul A. Goepfert

المصدر: Journal of Virology. 73:7210-7217

مصطلحات موضوعية: Cytoplasm, viruses, Immunology, Endoplasmic Reticulum, Microbiology, Cell Line, Dogs, Viral Envelope Proteins, Cricetinae, Virology, Animals, Humans, Spumavirus, Glycoproteins, Infectivity, chemistry.chemical_classification, Budding, biology, Lysine, KKXX, Endoplasmic reticulum, Intracellular Membranes, Human foamy virus, biology.organism_classification, Virus-Cell Interactions, chemistry, Insect Science, Glycoprotein

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::abd6580020fe2ee54b39d6009574359e
https://doi.org/10.1128/jvi.73.9.7210-7217.1999

المؤلفون: Hubert B. Gaspar, Fabio Candotti, Kimberly Gilmour, Alejandra Davila, Anna Paruzynski, Karen F. Buckland, Robert A. Sokolic, Denise Carbonaro, Kit L. Shaw, Claire Booth, Dayna Terrazs, Manfred G. Schmidt, Provobati Barman, Adrian J. Thrasher, Kenneth Cornetta, Donald B. Kohn

المصدر: Molecular Therapy. 23:S102-S103

مصطلحات موضوعية: Pharmacology, Severe combined immunodeficiency, Naive T cell, T cell, Genetic enhancement, Biology, medicine.disease, Viral vector, Adenosine deaminase deficiency, Haematopoiesis, medicine.anatomical_structure, Drug Discovery, Immunology, Genetics, medicine, Molecular Medicine, Stem cell, Molecular Biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::425c5e9bf05c3f6d2a6acb8780c599da
https://doi.org/10.1016/s1525-0016(16)33866-7

المؤلفون: Kit L. Shaw, Elizabeth Garabedian, Rob Sokolic, Provaboti Barman, Alejandra Davila, Christopher Silvin, Satiro de Oliveira, Ami J. Shah, Dayna Terrazas, Denise Carbonaro, Sabine Geiger, Suparna Mishra, Aaron Cooper, Monika Smogorzewska, Jayashree Jagadeesh, Michael S. Hershfield, Alan Wayne, Gay M. Crooks, Theodore Moore, Fabio Candotti, Donald B. Kohn

المصدر: Molecular Therapy. 23:S13-S14

مصطلحات موضوعية: Pharmacology, Chemotherapy, biology, business.industry, medicine.medical_treatment, Genetic enhancement, Phases of clinical research, Enzyme replacement therapy, Peripheral blood mononuclear cell, medicine.anatomical_structure, Immunology, Drug Discovery, medicine, biology.protein, Genetics, Molecular Medicine, Bone marrow, Antibody, business, Molecular Biology, Busulfan, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e3063675182e1f6bcda29c8f1571eb54

المؤلفون: Kit L. Shaw, Donald B. Kohn

المصدر: Science Translational Medicine. 3

مصطلحات موضوعية: Adenosine Deaminase, Genetic enhancement, Disease, X-Linked Combined Immunodeficiency Diseases, Adenosine deaminase, Agammaglobulinemia, medicine, Humans, Clinical Trials as Topic, Severe combined immunodeficiency, Myelosuppressive Chemotherapy, biology, business.industry, Hematopoietic Stem Cell Transplantation, Translational medicine, Infant, Hematopoietic stem cell, General Medicine, medicine.disease, United States, Transplantation, medicine.anatomical_structure, Italy, Immunology, biology.protein, Severe Combined Immunodeficiency, France, business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::01d7f0edcd7228303a8934eb62afdeba
https://doi.org/10.1126/scitranslmed.3002594