المؤلفون: Ragni, Margaret, Mead, Henry, de Jong, Ype, Kaczmarek, Radoslaw, Leavitt, Andrew, Long, Brian, Nugent, Diane, Sabatino, Denise, Fong, Sylvia, von Drygalski, Annette, Walsh, Christopher, Luxon, Bruce

المصدر: Blood Advances. 8(19)

مصطلحات موضوعية: Hemophilia A, Humans, Genetic Therapy, Liver, Dependovirus, Genetic Vectors, Factor VIII

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/0258w8c5
https://escholarship.org/content/qt0258w8c5/qt0258w8c5.pdf

المؤلفون: Mandal, Kamal, Wicaksono, Gianina, Yu, Clinton, Adams, Jarrett J, Hoopmann, Michael R, Temple, William C, Izgutdina, Adila, Escobar, Bonell Patiño, Gorelik, Maryna, Ihling, Christian H, Nix, Matthew A, Naik, Akul, Xie, William H, Hübner, Juwita, Rollins, Lisa A, Reid, Sandy M, Ramos, Emilio, Kasap, Corynn, Steri, Veronica, Serrano, Juan Antonio Camara, Salangsang, Fernando, Phojanakong, Paul, McMillan, Melanie, Gavallos, Victor, Leavitt, Andrew D, Logan, Aaron C, Rooney, Cliona M, Eyquem, Justin, Sinz, Andrea, Huang, Benjamin J, Stieglitz, Elliot, Smith, Catherine C, Moritz, Robert L, Sidhu, Sachdev S, Huang, Lan, Wiita, Arun P

المصدر: Nature Cancer. 4(11)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Oncology and Carcinogenesis, Immunology, Cancer, Hematology, Orphan Drug, Gene Therapy, Rare Diseases, Pediatric Cancer, Pediatric, Childhood Leukemia, Genetics, Immunotherapy, Biotechnology, 5.2 Cellular and gene therapies, 5.1 Pharmaceuticals, Humans, Receptors, Chimeric Antigen, T-Lymphocytes, Integrins, Immunotherapy, Adoptive, Leukemia, Myeloid, Acute, Oncology and carcinogenesis

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/9mr7d89n
https://escholarship.org/content/qt9mr7d89n/qt9mr7d89n.pdf

المؤلفون: Hu, Yang, Geere, Mimansa, Awan, Maham, Leavitt, Andrew D, Brown, Laura E, Pearson, Hadley J, Gandelman, Jocelyn S, Kogan, Scott C

المصدر: Hematology. 27(1)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Hematology, Clinical Research, Rare Diseases, 6.1 Pharmaceuticals, Evaluation of treatments and therapeutic interventions, Good Health and Well Being, Female, Humans, Glucosephosphate Dehydrogenase Deficiency, Methemoglobinemia, Dapsone, Hemolysis, Urticaria, Glucose-6-phosphate dehydrogenase, G6PD, bite cells, heinz bodies, methemoglobinemia, dapsone, dermatology, anemia, Cardiorespiratory Medicine and Haematology, Cardiovascular medicine and haematology

URL الوصول: https://escholarship.org/uc/item/5kx6t14t

المؤلفون: Cuker, Adam, Kavakli, Kaan, Frenzel, Laurent, Wang, Jiaan Der, Astermark, Jan, Cerqueira, Monica H., Iorio, Alfonso, Katsarou-Fasouli, Olga, Klamroth, Robert, Shapiro, Amy D., Hermans, Cédric, Ishiguro, Akira, Leavitt, Andrew D., Oldenburg, Johannes B., Ozelo, Margareth C., Teitel, Jerome, Biondo, Francesca, Fang, Annie, Fuiman, Joanne, Mckay, John, Sun, Pengling, Rasko, John E.J., Rupon, Jeremy

المصدر: New England Journal of Medicine. 391(12):1108-1118

مصطلحات موضوعية: Childhood Diseases, Coagulation, Genetics, Genetics General, Hematology/Oncology, Pediatrics, Medicin och hälsovetenskap, Klinisk medicin, Hematologi, Medical and Health Sciences, Clinical Medicine, Hematology

URL الوصول: https://lup.lub.lu.se/record/fa33668e-aa68-4926-bc83-2b5a3e3cf3f4
http://dx.doi.org/10.1056/NEJMoa2302982

المؤلفون: Valet, Colin, Magnen, Mélia, Qiu, Longhui, Cleary, Simon J, Wang, Kristin M, Ranucci, Serena, Grockowiak, Elodie, Boudra, Rafik, Conrad, Catharina, Seo, Yurim, Calabrese, Daniel R, Greenland, John R, Leavitt, Andrew D, Passegué, Emmanuelle, Mendez-Ferrer, Simon, Swirski, Filip K, Looney, Mark R

المصدر: Journal of Clinical Investigation. 132(7)

مصطلحات موضوعية: Hematology, Infectious Diseases, Sepsis, Underpinning research, 1.1 Normal biological development and functioning, Inflammatory and immune system, Cardiovascular, Blood, Good Health and Well Being, Animals, Blood Platelets, CD40 Ligand, Megakaryocytes, Spleen, Hematopoietic stem cells, Innate immunity, Platelets, Stem cells, Medical and Health Sciences, Immunology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/8bq955c8
https://escholarship.org/content/qt8bq955c8/qt8bq955c8.pdf

المؤلفون: Klamroth, Robert, Hayes, Gregory, Andreeva, Tatiana, Gregg, Keith, Suzuki, Takashi, Mitha, Ismail Haroon, Hardesty, Brandon, Shima, Midori, Pollock, Toni, Slev, Patricia, Oldenburg, Johannes, Ozelo, Margareth C, Stieltjes, Natalie, Castet, Sabine-Marie, Mahlangu, Johnny, Peyvandi, Flora, Kazmi, Rashid, Schved, Jean-François, Leavitt, Andrew D, Callaghan, Michael, Pan-Petesch, Brigitte, Quon, Doris V, Andrews, Jayson, Trinh, Alex, Li, Mingjin, Wong, Yen

المصدر: Human Gene Therapy. 33(7-8)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Genetics, Prevention, Antibodies, Neutralizing, Antibodies, Viral, Dependovirus, Genetic Vectors, Hemophilia A, Humans, Prospective Studies, Seroepidemiologic Studies, Serogroup, gene therapy, hemophilia A, adeno-associated virus, antibody, seropositivity, Clinical Sciences, Biotechnology, Medical biotechnology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/4fd5t2tv
https://escholarship.org/content/qt4fd5t2tv/qt4fd5t2tv.pdf

المؤلفون: Kennedy, Vanessa E, Wong, Chelsea C, Hong, Jessica M, Peng, Theodore An, Brondfield, Sam, Reilly, Linda M, Cornett, Patricia, Leavitt, Andrew D

المصدر: Blood Advances. 5(22)

مصطلحات موضوعية: Vaccine Related, Clinical Research, Hematology, Immunization, Biotechnology, Prevention, Good Health and Well Being, Ad26COVS1, COVID-19, COVID-19 Vaccines, Humans, Male, Thrombocytopenia, Thrombosis, Vaccination

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/21r8d0rd

المؤلفون: Feng, Qianxi, Nickels, Eric, Muskens, Ivo S, de Smith, Adam J, Gauderman, W James, Yee, Amy C, Ricker, Charite, Mack, Thomas, Leavitt, Andrew D, Godley, Lucy A, Wiemels, Joseph L

مصطلحات موضوعية: early-onset cancer, epidemiology, familial risk, global health, hispanic paradox, human, latino americans, linked cancer registry, race/ethnicity, Biochemistry and Cell Biology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/1r31w3fk

المؤلفون: Ragni, Margaret V., Mead, Henry, de Jong, Ype P., Kaczmarek, Radoslaw, Leavitt, Andrew D., Long, Brian, Nugent, Diane J., Sabatino, Denise E., Fong, Sylvia, von Drygalski, Annette, Walsh, Christopher E., Luxon, Bruce A.

المساهمون: Pediatrics, School of Medicine

المصدر: PMC

مصطلحات موضوعية: Dependovirus, Factor VIII, Genetic therapy, Hemophilia A, Liver

وصف الملف: application/pdf

Relation: Blood Advances; https://hdl.handle.net/1805/45012

الاتاحة: https://hdl.handle.net/1805/45012

المؤلفون: Madan, Bella, Ozelo, Margareth C., Raheja, Priyanka, Symington, Emily, Quon, Doris V., Leavitt, Andrew D., Pipe, Steven W., Lowe, Gillian, Kenet, Gili, Reding, Mark T., Mason, Jane, Wang, Michael, von Drygalski, Annette, Klamroth, Robert, Shapiro, Susan, Chambost, Hervé, Dunn, Amy L., Oldenburg, Johannes, Chou, Sheng-Chieh, Peyvandi, Flora, Millar, Carolyn M., Osmond, Dane, Yu, Hua, Dashiell-Aje, Ebony, Robinson, Tara M., Mahlangu, Johnny

المساهمون: B. Madan, M.C. Ozelo, P. Raheja, E. Symington, D.V. Quon, A.D. Leavitt, S.W. Pipe, G. Lowe, G. Kenet, M.T. Reding, J. Mason, M. Wang, A. von Drygalski, R. Klamroth, S. Shapiro, H. Chambost, A.L. Dunn, J. Oldenburg, S. Chou, F. Peyvandi, C.M. Millar, D. Osmond, Y. Hua, E. Dashiell-Aje, T.M. Robinson, J. Mahlangu

مصطلحات موضوعية: adeno-associated viru, clinical trial, gene therapy, health-related quality of life, hemophilia A, phase 3, Settore MED/09 - Medicina Interna, Settore MEDS-05/A - Medicina interna

Relation: info:eu-repo/semantics/altIdentifier/pmid/38614387; info:eu-repo/semantics/altIdentifier/wos/WOS:001263326800001; volume:22; issue:7; firstpage:1880; lastpage:1893; numberofpages:14; journal:JOURNAL OF THROMBOSIS AND HAEMOSTASIS; https://hdl.handle.net/2434/1076708

الاتاحة: https://hdl.handle.net/2434/1076708
https://doi.org/10.1016/j.jtha.2024.04.001

المؤلفون: Leavitt, Andrew D., Mahlangu, Johnny, Raheja, Priyanka, Symington, Emily, Quon, Doris V., Giermasz, Adam, López Fernández, Maria Fernanda, Kenet, Gili, Lowe, Gillian, Key, Nigel S., Millar, Carolyn M., Pipe, Steven W., Madan, Bella, Chou, Sheng-Chieh, Klamroth, Robert, Mason, Jane, Chambost, Hervé, Peyvandi, Flora, Majerus, Elaine, Pepperell, Dominic, Rivat, Christine, Yu, Hua, Robinson, Tara M., Ozelo, Margareth C.

المساهمون: BioMarin Pharmaceutical Inc

المصدر: Research and Practice in Thrombosis and Haemostasis ; volume 8, issue 8, page 102615 ; ISSN 2475-0379

الاتاحة: https://doi.org/10.1016/j.rpth.2024.102615
https://api.elsevier.com/content/article/PII:S2475037924003108?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S2475037924003108?httpAccept=text/plain

المؤلفون: Leaf, Rebecca Karp, Ferreri, Christopher, Rangachari, Deepa, Mier, James, Witteles, Wesley, Ansstas, George, Anagnostou, Theodora, Zubiri, Leyre, Piotrowska, Zofia, Oo, Thein H, Iberri, David, Yarchoan, Mark, Salama, April KS, Johnson, Douglas B, Leavitt, Andrew D, Rahma, Osama E, Reynolds, Kerry L, Leaf, David E

المصدر: American Journal of Hematology. 94(5)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Clinical Sciences, Immunology, Rare Diseases, Hematology, Autoimmune Disease, Adolescent, Adult, Aged, Aged, 80 and over, Anemia, Hemolytic, Autoimmune, Female, Glucocorticoids, Hemoglobins, Humans, Immunosuppression Therapy, Male, Middle Aged, Cardiorespiratory Medicine and Haematology, Cardiovascular medicine and haematology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/4rb5x8rb

المؤلفون: Chao, Hui Xiao, Zack, Travis, Leavitt, Andrew D.

المصدر: Obstetrics & Gynecology; Jan2025, Vol. 145 Issue 1, p91-94, 4p

المؤلفون: Mahlangu, Johnny, Kaczmarek, Radoslaw, von Drygalski, Annette, Shapiro, Susan, Chou, Sheng-Chieh, Ozelo, Margareth C, Kenet, Gili, Peyvandi, Flora, Wang, Michael, Madan, Bella, Key, Nigel S, Laffan, Michael, Dunn, Amy L, Mason, Jane, Quon, Doris V, Symington, Emily, Leavitt, Andrew D, Oldenburg, Johannes, Chambost, Hervé, Reding, Mark T, Jayaram, Kala, Yu, Hua, Mahajan, Reena, Chavele, Konstantia-Maria, Reddy, Divya B, Henshaw, Joshua, Robinson, Tara M, Wong, Wing Yen, Pipe, Steven W, GENEr8-1 Trial Group, Hermans, Cédric

المساهمون: UCL - SSS/IREC/CARD - Pôle de recherche cardiovasculaire, UCL - (SLuc) Service d'hématologie, UCL - (SLuc) Centre de malformations vasculaires congénitales

المصدر: The New England journal of medicine, Vol. 388, no.8, p. 694-705 (2023)

مصطلحات موضوعية: Humans, Male, Factor VIII, Gene Transfer Techniques, Half-Life, Hemophilia A, Hemorrhage, Recombinant Fusion Proteins

Relation: boreal:274106; http://hdl.handle.net/2078.1/274106; info:pmid/36812433; urn:ISSN:0028-4793; urn:EISSN:1533-4406

الاتاحة: http://hdl.handle.net/2078.1/274106
https://doi.org/10.1056/NEJMoa2211075

المؤلفون: Ragni, Margaret V., Callis, Jessica, Daoud, Nabil, Hu, Brian, Manuel, Matthew, Santos, Jarren, Schwartz, Jonathan, Friedman, Kenneth D., Kouides, Peter, Kuriakose, Philip, Leavitt, Andrew D., Lim, Ming Y., Machin, Nicoletta, Recht, Michael, Chrisentery‐Singleton, Tammuella

المساهمون: BioMarin Pharmaceutical, Pennsylvania Department of Health

المصدر: Haemophilia ; volume 30, issue 1, page 87-97 ; ISSN 1351-8216 1365-2516

الاتاحة: http://dx.doi.org/10.1111/hae.14910
https://onlinelibrary.wiley.com/doi/pdf/10.1111/hae.14910

المؤلفون: Ragni, Margaret V, Rothenberger, Scott D, Feldman, Robert, Nance, Danielle, Leavitt, Andrew D, Malec, Lynn, Kulkarni, Roshni, Sidonio, Robert, Kraut, Eric, Lasky, Joseph, Pruthi, Rajiv, Angelini, Dana, Philipp, Claire, Hwang, Nina, Wheeler, Allison P, Seaman, Craig, Machin, Nicoletta, Xavier, Frederico, Meyer, Michael, Bellissimo, Daniel, Humphreys, Gregory, Smith, Kenneth J, Merricks, Elizabeth P, Nichols, Timothy C, Ivanco, Dana, Vehec, Deborah, Koerbel, Glory, Althouse, Andrew D

المصدر: The Lancet Haematology ; volume 10, issue 8, page e612-e623 ; ISSN 2352-3026

الاتاحة: http://dx.doi.org/10.1016/s2352-3026(23)00119-9
https://api.elsevier.com/content/article/PII:S2352302623001199?httpAccept=text/xml
https://api.elsevier.com/content/article/PII:S2352302623001199?httpAccept=text/plain

المؤلفون: Lefrançais, Emma, Ortiz-Muñoz, Guadalupe, Caudrillier, Axelle, Mallavia, Beñat, Liu, Fengchun, Sayah, David M, Thornton, Emily E, Headley, Mark B, David, Tovo, Coughlin, Shaun R, Krummel, Matthew F, Leavitt, Andrew D, Passegué, Emmanuelle, Looney, Mark R

المصدر: Nature. 544(7648)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Cardiovascular Medicine and Haematology, Stem Cell Research, Lung Cancer, Lung, Cancer, Hematology, Animals, Blood Platelets, Bone Marrow, Cell Lineage, Female, Hematopoiesis, Hematopoietic Stem Cells, Male, Megakaryocytes, Mice, Microcirculation, Platelet Count, Thrombocytopenia, General Science & Technology

وصف الملف: application/pdf

URL الوصول: https://escholarship.org/uc/item/1mg1s5kf
https://escholarship.org/content/qt1mg1s5kf/qt1mg1s5kf.pdf

المؤلفون: Goyal, Vanshika, Salmasi, Giselle, Leavitt, Andrew D., Rubenstein, James L., Banerjee, Rahul

المصدر: eJHaem ; volume 3, issue 3, page 1000-1002 ; ISSN 2688-6146 2688-6146

الاتاحة: http://dx.doi.org/10.1002/jha2.482
https://onlinelibrary.wiley.com/doi/pdf/10.1002/jha2.482
https://onlinelibrary.wiley.com/doi/full-xml/10.1002/jha2.482

المؤلفون: Payne, Jeanette, Batsuli, Glaivy, Leavitt, Andrew D., Mathias, Mary, McGuinn, Catherine E.

المصدر: Haemophilia ; volume 28, issue 4, page 523-531 ; ISSN 1351-8216 1365-2516

الاتاحة: http://dx.doi.org/10.1111/hae.14570
https://onlinelibrary.wiley.com/doi/pdf/10.1111/hae.14570
https://onlinelibrary.wiley.com/doi/full-xml/10.1111/hae.14570

المؤلفون: Leavitt, Andrew S., Nelson, Kari L., Cutucache, Christine E.

المصدر: Frontiers in Education ; volume 6 ; ISSN 2504-284X

الاتاحة: http://dx.doi.org/10.3389/feduc.2021.731657
https://www.frontiersin.org/articles/10.3389/feduc.2021.731657/full