المؤلفون: Cornetta, Kenneth, Yao, Jing, House, Kimberley, Duffy, Lisa, Adusumilli, Prasad, Beyer, Rachel, Booth, Claire, Brenner, Malcolm, Curran, Kevin, Grilley, Bambi, Heslop, Helen, Hinrichs, Christian, Kaplan, Rosandra, Kiem, Hans-Peter, Kochenderfer, James, Rivière, Isabelle, Rosenberg, Steven, Sauter, Craig, Shah, Nirali, Slovin, Susan, Thrasher, Adrian, Williams, David, Lin, Tsai-Yu, Mailankody, Sham, Norberg, Scott, OCearbhaill, Roisin, Pappas, Jennifer, Park, Jae, Ramos, Carlos, Kohn, Donald, Ribas, Antoni

المصدر: Molecular Therapy. 31(3)

مصطلحات موضوعية: cancer immunotherapy, gene therapy, patient monitoring, replication competent retrovirus, safety, Humans, Virus Replication, Retroviridae, Genetic Vectors, Cell Line, Genetic Therapy

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/59d673fcTest

المؤلفون: Bahal, Sameer, Zinicola, Marta, Moula, Shefta E, Whittaker, Thomas E, Schejtman, Andrea, Naseem, Asma, Blanco, Elena, Vetharoy, Winston, Hu, Yi-Ting, Rai, Rajeev, Gomez-Castaneda, Eduardo, Cunha Santos, Catarina, Burns, Siobhan O, Morris, Emma C, Booth, Claire, Turchiano, Giandomenico, Cavazza, Alessia, Thrasher, Adrian J, Santilli, Giorgia

المصدر: Journal of Allergy and Clinical Immunology (2024) (In press).

مصطلحات موضوعية: B cell, agammaglobulinemia, gene editing, hematopoietic stem and progenitor cells

وصف الملف: text

العلاقة: https://discovery.ucl.ac.uk/id/eprint/10189377/1/1-s2.0-S0091674924002409-main.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10189377Test/

الإتاحة: https://discovery.ucl.ac.uk/id/eprint/10189377/1/1-s2.0-S0091674924002409-main.pdfTest
https://discovery.ucl.ac.uk/id/eprint/10189377Test/

المؤلفون: Rai, Rajeev, Steinberg, Zohar, Romito, Marianna, Zinghirino, Federica, Hu, Yi-Ting, White, Nathan, Naseem, Asma, Thrasher, Adrian J, Turchiano, Giandomenico, Cavazza, Alessia

المصدر: Human Gene Therapy (2024) (In press).

مصطلحات موضوعية: Gene editing, hematopoietic stem cells, T cells, immunodeficiency, disease modelling

وصف الملف: text

العلاقة: https://discovery.ucl.ac.uk/id/eprint/10186154/1/rai-et-al-2024-crispr-cas9-based-disease-modelling-and-functional-correction-of-interleukin-7-receptor-alpha-severe.pdfTest; https://discovery.ucl.ac.uk/id/eprint/10186154Test/

الإتاحة: https://discovery.ucl.ac.uk/id/eprint/10186154/1/rai-et-al-2024-crispr-cas9-based-disease-modelling-and-functional-correction-of-interleukin-7-receptor-alpha-severe.pdfTest
https://discovery.ucl.ac.uk/id/eprint/10186154Test/

المؤلفون: Vaidyanathan, Sriram, Kerschner, Jenny L., Paranjapye, Alekh, Sinha, Vrishti, Lin, Brian, Bedrosian, Tracy A., Thrasher, Adrian J., Turchiano, Giandomenico, Harris, Ann, Porteus, Matthew H.

المساهمون: Cystic Fibrosis Foundation, Wellcome Trust, National Institutes of Health

المصدر: Molecular Therapy - Nucleic Acids ; volume 35, issue 1, page 102134 ; ISSN 2162-2531

مصطلحات موضوعية: Drug Discovery, Molecular Medicine

الإتاحة: https://doi.org/10.1016/j.omtn.2024.102134Test
https://api.elsevier.com/content/article/PII:S2162253124000210?httpAccept=text/xmlTest
https://api.elsevier.com/content/article/PII:S2162253124000210?httpAccept=text/plainTest

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

المصدر: New England Journal of Medicine. 384(21)

مصطلحات موضوعية: Medical Biotechnology, Biomedical and Clinical Sciences, Biotechnology, Transplantation, Gene Therapy, Genetics, Regenerative Medicine, Clinical Research, Stem Cell Research, Stem Cell Research - Nonembryonic - Human, Evaluation of treatments and therapeutic interventions, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, 6.2 Cellular and gene therapies, Good Health and Well Being, Adenosine Deaminase, Adolescent, Agammaglobulinemia, Child, Child, Preschool, Genetic Therapy, Genetic Vectors, Hematopoietic Stem Cell Transplantation, Humans, Infant, Lentivirus, Lymphocyte Count, Progression-Free Survival, Prospective Studies, Severe Combined Immunodeficiency, Transplantation, Autologous, Medical and Health Sciences, General & Internal Medicine, Biomedical and clinical sciences, Health sciences

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/0tw1k4mmTest

المؤلفون: Kohn, Donald B, Booth, Claire, Kang, Elizabeth M, Pai, Sung-Yun, Shaw, Kit L, Santilli, Giorgia, Armant, Myriam, Buckland, Karen F, Choi, Uimook, De Ravin, Suk See, Dorsey, Morna J, Kuo, Caroline Y, Leon-Rico, Diego, Rivat, Christine, Izotova, Natalia, Gilmour, Kimberly, Snell, Katie, Dip, Jinhua Xu-Bayford, Darwish, Jinan, Morris, Emma C, Terrazas, Dayna, Wang, Leo D, Bauser, Christopher A, Paprotka, Tobias, Kuhns, Douglas B, Gregg, John, Raymond, Hayley E, Everett, John K, Honnet, Geraldine, Biasco, Luca, Newburger, Peter E, Bushman, Frederic D, Grez, Manuel, Gaspar, H Bobby, Williams, David A, Malech, Harry L, Galy, Anne, Thrasher, Adrian J, Net4CGD consortium

المصدر: Nature medicine. 26(2)

مصطلحات موضوعية: Net4CGD consortium, Neutrophils, Hematopoietic Stem Cells, Chromosomes, Human, X, Humans, Lentivirus, Granulomatous Disease, Chronic, Antigens, CD34, Treatment Outcome, Transplantation Conditioning, Comorbidity, Gene Silencing, Genes, Regulator, Genetic Vectors, Adolescent, Child, Child, Preschool, United States, Male, Promoter Regions, Genetic, Young Adult, Patient Safety, Genetic Therapy, United Kingdom, NADPH Oxidases, Genetics, Hematology, Regenerative Medicine, Stem Cell Research, Stem Cell Research - Nonembryonic - Human, Gene Therapy, Clinical Trials and Supportive Activities, Infectious Diseases, Clinical Research, 5.2 Cellular and gene therapies, Development of treatments and therapeutic interventions, Inflammatory and immune system, Infection, Medical and Health Sciences, Immunology

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/7b61w03mTest

المؤلفون: Sereni, Lucia, Castiello, Maria Carmina, Marangoni, Francesco, Anselmo, Achille, di Silvestre, Dario, Motta, Sara, Draghici, Elena, Mantero, Stefano, Thrasher, Adrian J, Giliani, Silvia, Aiuti, Alessandro, Mauri, Pierluigi, Notarangelo, Luigi D, Bosticardo, Marita, Villa, Anna

المصدر: Journal of Allergy and Clinical Immunology. 142(4)

مصطلحات موضوعية: Biomedical and Clinical Sciences, Immunology, Clinical Research, Rare Diseases, 2.1 Biological and endogenous factors, Aetiology, Inflammatory and immune system, Adolescent, Adult, Animals, Autoimmunity, Blood Platelets, CD40 Ligand, Child, Child, Preschool, Female, Humans, Infant, Inflammation, Mice, Inbred C57BL, Mice, Knockout, Platelet Count, Wiskott-Aldrich Syndrome, Wiskott-Aldrich Syndrome Protein, Young Adult, Wiskott-Aldrich syndrome, platelet deficiency, CD40 ligand, autoantibodies, autoimmunity, Allergy

وصف الملف: application/pdf

الوصول الحر: https://escholarship.org/uc/item/2xp338x4Test

المؤلفون: Westhaus, Adrian, Cabanes-Creus, Marti, Dilworth, Kimberley L., Zhu, Erhua, Salas Gómez, David, Navarro, Renina G., Amaya, Anais K., Scott, Suzanne, Kwiatek, Magdalena, McCorkindale, Alexandra L., Hayman, Tara E., Frahm, Silke, Perocheau, Dany P., Tran, Bang Manh, Vincan, Elizabeth, Wong, Sharon L., Waters, Shafagh A., Riddiough, Georgina E., Perini, Marcos V., Wilson, Laurence O. W., Baruteau, Julien, Diecke, Sebastian, González-Aseguinolaza, Gloria, Santilli, Giorgia, Thrasher, Adrian J., Alexander, Ian E., Lisowski, Leszek

المصدر: Westhaus , A , Cabanes-Creus , M , Dilworth , K L , Zhu , E , Salas Gómez , D , Navarro , R G , Amaya , A K , Scott , S , Kwiatek , M , McCorkindale , A L , Hayman , T E , Frahm , S , Perocheau , D P , Tran , B M , Vincan , E , Wong , S L , Waters , S A , Riddiough , G E , Perini , M V , Wilson , L O W , Baruteau , J , Diecke ....

مصطلحات موضوعية: AAV, hepatocyte, liver gene therapy, nonhuman primate, xenograft model

وصف الملف: application/pdf

الإتاحة: https://doi.org/10.1089/hum.2022.188Test
https://researchers.mq.edu.au/en/publications/95099273-7274-4f80-a056-d9d923d09602Test
https://research-management.mq.edu.au/ws/files/345499599/341430772.pdfTest
http://www.scopus.com/inward/record.url?scp=85153410519&partnerID=8YFLogxKTest
http://purl.org/au-research/grants/arc/DP150101253Test
http://purl.org/au-research/grants/nhmrc/1108311Test

المؤلفون: Minskaia, Ekaterina, Maimaris, Jesmeen, Jenkins, Persephone, Albuquerque, Adriana S., Hong, Ying, Eleftheriou, Despina, Gilmour, Kimberly C., Grace, Richard, Moreira, Fernando, Grimbacher, Bodo, Adhya, Zoe, Alachkar, Hana, Anantharachagan, Ariharan, Antrobus, Richard, Arumugakani, Gururaj, Bacchelli, Chiara, Baxendale, Helen, Bethune, Claire, Bibi, Shahnaz, Boardman, Barbara, Booth, Claire, Browning, Michael, Brownlie, Mary, Burns, Siobhan, Chandra, Anita, Clifford, Hayley, Cooper, Nichola, Davies, Sophie, Dempster, John, Devlin, Lisa, Doffinger, Rainer, Drewe, Elizabeth, Edgar, David, Egner, William, El-Shanawany, Tariq, Gaspar, Bobby, Ghurye, Rohit, Gilmour, Kimberley, Goddard, Sarah, Gordins, Pavel, Grigoriadou, Sofia, Hackett, Scott, Hague, Rosie, Harper, Lorraine, Hayman, Grant, Herwadkar, Archana, Hughes, Stephen, Huissoon, Aarnoud, Jolles, Stephen, Jones, Julie, Kelleher, Peter, Klein, Nigel, Kuijpers, Taco, Kumararatne, Dinakantha, Laffan, James, Allen, Hana Lango, Lear, Sara, Longhurst, Hilary, Lorenzo, Lorena, Manson, Ania, McDermott, Elizabeth, Millar, Hazel, Mistry, Anoop, Morrisson, Valerie, Murng, Sai, Nasir, Iman, Nejentsev, Sergey, Noorani, Sadia, Oksenhendler, Eric, Ponsford, Mark, Qasim, Waseem, Quinn, Ellen, Quinti, Isabella, Richter, Alex, Samarghitean, Crina, Sargur, Ravishankar, Savic, Sinisa, Seneviratne, Suranjith, Sewall, Carrock, Shackley, Fiona, Simeoni, Ilenia, Smith, Kenneth G. C., Staples, Emily, Stauss, Hans, Steele, Cathal, Thaventhiran, James, Thomas, Moira, Thrasher, Adrian, Welch, Steve, Willcocks, Lisa, Workman, Sarita, Worth, Austen, Yeatman, Nigel, Yong, Patrick, Ashford, Sofie, Bradley, John, Fletcher, Debra, Hammerton, Tracey, James, Roger, Kingston, Nathalie, Ouwehand, Willem, Penkett, Christopher, Raymond, F. Lucy, Stirrups, Kathleen, Veltman, Marijke, Young, Tim, Brown, Matthew, Clements-Brod, Naomi, Davis, John, Dewhurst, Eleanor, Erwood, Marie, Frary, Amy, Linger, Rachel, Martin, Jennifer, Papadia, Sofia, Rehnstrom, Karola, Astle, William, Attwood, Antony, Bleda, Marta, Carss, Keren, Daugherty, Louise, Deevi, Sri, Graf, Stefan, Greene, Daniel, Halmagyi, Csaba, Haimel, Matthias, Hu, Fengyuan, Matser, Vera, Meacham, Stuart, Megy, Karyn, Shamardina, Olga, Titterton, Catherine, Tuna, Salih, Turro, Ernest, Yu, Ping, von Ziegenweldt, Julie, Furnell, Abigail, Mapeta, Rutendo, Staines, Simon, Stephens, Jonathan, Whitehorn, Deborah, Rayner-Matthews, Paula, Watt, Christopher, Morris, Emma C., Burns, Siobhan O.

وصف الملف: application/pdf

العلاقة: https://orca.cardiff.ac.uk/id/eprint/160564/1/s10875-023-01530-7.pdfTest; Minskaia, Ekaterina, Maimaris, Jesmeen, Jenkins, Persephone, Albuquerque, Adriana S., Hong, Ying, Eleftheriou, Despina, Gilmour, Kimberly C., Grace, Richard, Moreira, Fernando, Grimbacher, Bodo, Adhya, Zoe, Alachkar, Hana, Anantharachagan, Ariharan, Antrobus, Richard, Arumugakani, Gururaj, Bacchelli, Chiara, Baxendale, Helen, Bethune, Claire, Bibi, Shahnaz, Boardman, Barbara, Booth, Claire, Browning, Michael, Brownlie, Mary, Burns, Siobhan, Chandra, Anita, Clifford, Hayley, Cooper, Nichola, Davies, Sophie, Dempster, John, Devlin, Lisa, Doffinger, Rainer, Drewe, Elizabeth, Edgar, David, Egner, William, El-Shanawany, Tariq https://orca.cardiff.ac.uk/view/cardiffauthors/A0388095.htmlTest, Gaspar, Bobby, Ghurye, Rohit, Gilmour, Kimberley, Goddard, Sarah, Gordins, Pavel, Grigoriadou, Sofia, Hackett, Scott, Hague, Rosie, Harper, Lorraine, Hayman, Grant, Herwadkar, Archana, Hughes, Stephen, Huissoon, Aarnoud, Jolles, Stephen https://orca.cardiff.ac.uk/view/cardiffauthors/A076912M.htmlTest, Jones, Julie, Kelleher, Peter, Klein, Nigel, Kuijpers, Taco, Kumararatne, Dinakantha, Laffan, James, Allen, Hana Lango, Lear, Sara, Longhurst, Hilary, Lorenzo, Lorena, Maimaris, Jesmeen, Manson, Ania, McDermott, Elizabeth, Millar, Hazel, Mistry, Anoop, Morrisson, Valerie, Murng, Sai, Nasir, Iman, Nejentsev, Sergey, Noorani, Sadia, Oksenhendler, Eric, Ponsford, Mark, Qasim, Waseem, Quinn, Ellen, Quinti, Isabella, Richter, Alex, Samarghitean, Crina, Sargur, Ravishankar, Savic, Sinisa, Seneviratne, Suranjith, Sewall, Carrock, Shackley, Fiona, Simeoni, Ilenia, Smith, Kenneth G. C., Staples, Emily, Stauss, Hans, Steele, Cathal, Thaventhiran, James, Thomas, Moira, Thrasher, Adrian, Welch, Steve, Willcocks, Lisa, Workman, Sarita, Worth, Austen, Yeatman, Nigel, Yong, Patrick, Ashford, Sofie, Bradley, John, Fletcher, Debra, Hammerton, Tracey, James, Roger, Kingston, Nathalie, Ouwehand, Willem, Penkett, Christopher, Raymond, F. Lucy, Stirrups, Kathleen, Veltman, Marijke, Young, Tim, Brown, Matthew, Clements-Brod, Naomi, Davis, John, Dewhurst, Eleanor, Erwood, Marie, Frary, Amy, Linger, Rachel, Martin, Jennifer, Papadia, Sofia, Rehnstrom, Karola, Astle, William, Attwood, Antony, Bleda, Marta, Carss, Keren, Daugherty, Louise, Deevi, Sri, Graf, Stefan, Greene, Daniel, Halmagyi, Csaba, Haimel, Matthias, Hu, Fengyuan, Matser, Vera, Meacham, Stuart, Megy, Karyn, Shamardina, Olga, Titterton, Catherine, Tuna, Salih, Turro, Ernest, Yu, Ping, von Ziegenweldt, Julie, Furnell, Abigail, Mapeta, Rutendo, Staines, Simon, Stephens, Jonathan, Whitehorn, Deborah, Rayner-Matthews, Paula, Watt, Christopher, Morris, Emma C. and Burns, Siobhan O. 2023. Autosomal dominant STAT6 Gain of function causes severe atopy associated with lymphoma. Journal of Clinical Immunology 10.1007/s10875-023-01530-7 https://doi.org/10.1007/s10875-023-01530-7Test file https://orca.cardiff.ac.uk/id/eprint/160564/1/s10875-023-01530-7.pdfTest

الإتاحة: https://doi.org/10.1007/s10875-023-01530-7Test
https://orca.cardiff.ac.uk/id/eprint/160564Test/
https://orca.cardiff.ac.uk/id/eprint/160564/1/s10875-023-01530-7.pdfTest

المؤلفون: Rae, William, Sowerby, John M, Verhoeven, Dorit, Youssef, Mariam, Kotagiri, Prasanti, Savinykh, Natalia, Coomber, Eve L, Boneparth, Alexis, Chan, Angela, Gong, Chun, Jansen, Machiel H, du Long, Romy, Santilli, Giorgia, Simeoni, Ilenia, Stephens, Jonathan, Wu, Kejia, Zinicola, Marta, Allen, Hana Lango, Baxendale, Helen, Kumararatne, Dinakantha, Gkrania-Klotsas, Effrossyni, Scheffler Mendoza, Selma C, Yamazaki-Nakashimada, Marco Antonio, Ruiz, Laura Berrón, Rojas-Maruri, Cesar Mauricio, Lugo Reyes, Saul O, Lyons, Paul A, Williams, Anthony P, Hodson, Daniel J, Bishop, Gail A, Thrasher, Adrian J, Thomas, David C, Murphy, Michael P, Vyse, Timothy J, Milner, Joshua D, Kuijpers, Taco W, Smith, Kenneth GC

مصطلحات موضوعية: Autoimmunity, B-Lymphocytes, Humans, Mutation, Neoplasms, TNF Receptor-Associated Factor 3

وصف الملف: Print-Electronic; application/octet-stream; image/png

العلاقة: https://www.repository.cam.ac.uk/handle/1810/346347Test

الإتاحة: https://doi.org/10.17863/CAM.93768Test
https://www.repository.cam.ac.uk/handle/1810/346347Test