المؤلفون: Sarracino, Denise Carbonaro, Tarantal, Alice F, Lee, C Chang I, Martinez, Michele, Jin, Xiangyang, Wang, Xiaoyan, Hardee, Cinnamon L, Geiger, Sabine, Kahl, Christoph A, Kohn, Donald B

المصدر: Molecular Therapy. 22(10)

مصطلحات موضوعية: Medical Biotechnology, Medical Microbiology, Biomedical and Clinical Sciences, Infectious Diseases, Genetics, Rare Diseases, Gene Therapy, Transplantation, Biotechnology, HIV/AIDS, Sexually Transmitted Infections, Hematology, Pediatric, 5.2 Cellular and gene therapies, Good Health and Well Being, Adenosine Deaminase, Animals, Disease Models, Animal, Female, Gene Expression, Gene Order, Gene Transfer Techniques, Genetic Vectors, Humans, Lentivirus, Macaca mulatta, Mice, Mice, Knockout, Retroviridae, Severe Combined Immunodeficiency, Tissue Distribution, Transduction, Genetic, Transgenes, Biological Sciences, Technology, Medical and Health Sciences, Clinical sciences, Medical biotechnology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/4zr1j58r
https://escholarship.org/content/qt4zr1j58r/qt4zr1j58r.pdf

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: Frederick L Hall, Paul D Benya, Silvia R Padilla, Denise Carbonaro-Hall, Richard Williams, Sue Buckley, David Warburton, S A, ‡j Vernon

المساهمون: The Pennsylvania State University CiteSeerX Archives

المصدر: http://publicationslist.org/data/richard.williams/ref-33/1996-Hall-TGF%20Beta%20Receptor-BIOCHEM%20J.pdf.

وصف الملف: application/pdf

Relation: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.1037.9103; http://publicationslist.org/data/richard.williams/ref-33/1996-Hall-TGF%20Beta%20Receptor-BIOCHEM%20J.pdf

الاتاحة: http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.1037.9103
http://publicationslist.org/data/richard.williams/ref-33/1996-Hall-TGF%20Beta%20Receptor-BIOCHEM%20J.pdf

المؤلفون: Misako Kawahara, Andrea D. Karlen, Axel Schambach, Divya Punwani, Jason Yu, Kenneth Cornetta, Denise Carbonaro, Harry L. Malech, Michael Rothe, Sara Khan, Morton J. Cowan, Sushmita Roy, R. Scott McIvor, Jennifer M. Puck, Donald B. Kohn, Ukina Sanford, Kiran Patel

المصدر: HUMAN GENE THERAPY, vol 28, iss 1
Punwani, D; Kawahara, M; Yu, J; Sanford, U; Roy, S; Patel, K; et al.(2017). Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency. HUMAN GENE THERAPY, 28(1), 112-124. doi: 10.1089/hum.2016.064. UCSF: Retrieved from: http://www.escholarship.org/uc/item/8h27g83n

مصطلحات موضوعية: 0301 basic medicine, DNA Repair, DCLRE1C, T-Lymphocytes, Genetic enhancement, medicine.medical_treatment, Medical Biotechnology, Mice, SCID, Hematopoietic stem cell transplantation, Radiation Tolerance, Artemis, Mice, 0302 clinical medicine, lentivirus, Cells, Cultured, Research Articles, Mice, Knockout, B-Lymphocytes, Hematopoietic Stem Cell Transplantation, Nuclear Proteins, radiation sensitivity, severe combined immunodeficiency, Combined Modality Therapy, gene therapy, DNA-Binding Proteins, medicine.anatomical_structure, Molecular Medicine, Stem cell, Biotechnology, T cell, Genetic Vectors, Clinical Sciences, Biology, 03 medical and health sciences, Genetics, medicine, Animals, Humans, Molecular Biology, B cell, Severe combined immunodeficiency, Lentivirus, Genetic Therapy, Fibroblasts, Endonucleases, Hematopoietic Stem Cells, medicine.disease, Transplantation, Disease Models, Animal, 030104 developmental biology, Gamma Rays, Immunology, Severe Combined Immunodeficiency, 030215 immunology

وصف الملف: application/pdf

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::21b4424b89f69b924347691269aca783
https://escholarship.org/uc/item/8h27g83n

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: Sarah N. Ur, Celine J. Rocca, Laura Hernandez, Tatiana Lobry, Stephanie Cherqui, Denise Carbonaro, Athena Lau, Jay Sharma, Donald B. Kohn

المصدر: Molecular Therapy. 24:S14-S15

مصطلحات موضوعية: Pharmacology, Cystine, medicine.disease, Transplantation, chemistry.chemical_compound, Cystinosin, chemistry, Nephropathic Cystinosis, Drug Discovery, Cystinosis, Immunology, Genetics, medicine, Molecular Medicine, Autologous transplantation, Cysteamine, Thyroid function, Molecular Biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::73a28542a1d13add0980371c7f232b39
https://doi.org/10.1016/s1525-0016(16)32841-6

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: Jason Yu, Divya Punwani, Jennifer M. Puck, Donald B. Kohn, Scott McIvor, Misako Stillion, Harry L. Malech, Denise Carbonaro, Morton J. Cowan

المصدر: Molecular Therapy. 23:S117-S118

مصطلحات موضوعية: Pharmacology, Severe combined immunodeficiency, DCLRE1C, Lymphocyte, CD34, Biology, medicine.disease, Virology, Haematopoiesis, medicine.anatomical_structure, Drug Discovery, Cancer research, medicine, Genetics, Molecular Medicine, Progenitor cell, Stem cell, Molecular Biology, B cell

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::3de4ab42010c637e67b6e307709edb11

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: Athena Lau, Denise Carbonaro, Jay Sharma, Celine J. Rocca, Betty L. Cabrera, Sarah N. Ur, Donald B. Kohn, Tatiana Lobry, Stephanie Cherqui

المصدر: Molecular Therapy. 23:S36-S37

مصطلحات موضوعية: Pharmacology, Genetic enhancement, medicine.medical_treatment, Hematopoietic stem cell transplantation, Biology, medicine.disease, Transplantation, chemistry.chemical_compound, chemistry, Immunology, Cystinosis, Drug Discovery, Cancer research, medicine, Genetics, Autologous transplantation, Molecular Medicine, Cysteamine, Progenitor cell, Stem cell, Molecular Biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::38dea92cc3c47fed72fd4946931abbc2

المؤلفون: Laura Hernandez, Jay Sharma, Athena Lau, Stephanie Cherqui, Betty L. Cabrera, Sarah N. Ur, Tatiana Lobry, Denise Carbonaro, Celine J. Rocca, Donald B. Kohn

المصدر: Molecular Genetics and Metabolism. 120:S86

مصطلحات موضوعية: Oncology, medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Phases of clinical research, medicine.disease, Biochemistry, Transplantation, Haematopoiesis, Endocrinology, Internal medicine, Cystinosis, Genetics, medicine, Stem cell, business, Molecular Biology

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_________::5256bef3302c586b6a8c81f66007b830
https://doi.org/10.1016/j.ymgme.2016.11.209

المؤلفون: Yang Zhou, Michael R. Blackburn, David W. Gjertson, Michael L. Kaufman, Xiangyang Jin, Xingchao Wang, Xiaoyan Wang, Nora Rozengurt, Donald B. Kohn, Xiao-Jin Yu, Denise Carbonaro

مصطلحات موضوعية: medicine.medical_specialty, congenital, hereditary, and neonatal diseases and abnormalities, Transplantation Conditioning, Adenosine Deaminase, medicine.medical_treatment, Genetic enhancement, Genetic Vectors, Immunology, Urology, CD34, Hematopoietic stem cell transplantation, Biochemistry, Mice, Agammaglobulinemia, Transduction, Genetic, Animals, Medicine, Autologous transplantation, Bone Marrow Transplantation, Mice, Knockout, Severe combined immunodeficiency, business.industry, Hematopoietic Stem Cell Transplantation, nutritional and metabolic diseases, Genetic Therapy, Cell Biology, Hematology, Enzyme replacement therapy, Gene Therapy, medicine.disease, Adenosine deaminase deficiency, Disease Models, Animal, Retroviridae, Severe Combined Immunodeficiency, business

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b1daac350382dbfcb53a802b86dea13c
https://europepmc.org/articles/PMC3488883/

المؤلفون: Elizabeth Garabedian, Satiro N. De Oliveira, Kit L. Shaw, Denise Carbonaro, Alejandar Davila, Provaboti Barman, Christopher Silvin, Donald B. Kohn, Theodore B. Moore, Michael S. Hershfield, Ami J. Shah, H. Robert Gaspar, Adrian J. Thrasher, Fabio Candotti, Robert A. Sokolic

المصدر: ResearcherID

مصطلحات موضوعية: Transplantation, medicine.medical_specialty, Cyclophosphamide, business.industry, Genetic enhancement, medicine.medical_treatment, Immunosuppression, Hematology, Defibrotide, medicine.disease, Gastroenterology, Tacrolimus, Fludarabine, Calcineurin, surgical procedures, operative, Graft-versus-host disease, Internal medicine, medicine, business, medicine.drug

URL الوصول: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c91e266f9072dcf0e459592a1d56aa90
https://doi.org/10.1016/j.bbmt.2014.11.123