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1.

Differences in CD80 and CD86 transendocytosis reveal CD86 as a key target for CTLA-4 immune regulation.

Kennedy, Alan; Waters, Erin; Rowshanravan, Behzad; Hinze, Claudia; Williams, Cayman; Janman, Daniel; Fox, Thomas A; Booth, Claire; Pesenacker, Anne M; Halliday, Neil; Soskic, Blagoje; Kaur, Satdip; Qureshi, Omar S; Morris, Emma C; Ikemizu, Shinji; Paluch, Christopher; Huo, Jiandong; Davis, Simon J; Boucrot, Emmanuel; Walker, Lucy S K; Sansom, David M.

Nat Immunol ; 23(9): 1365-1378, 2022 09.

Artículo en Inglés | MEDLINE | ID: mdl-35999394

2.

Anti-CD45 PBD-based antibody-drug conjugates are effective targeted conditioning agents for gene therapy and stem cell transplant.

Yeung, Jenny; Liao, Aiyin; Shaw, Matthew; Silva, Soraia; Vetharoy, Winston; Rico, Diego Leon; Kirby, Ian; Zammarchi, Francesca; Havenith, Karin; de Haan, Lolke; van Berkel, Patrick H; Sebire, Neil; Ogunbiyi, Olumide K; Booth, Claire; Gaspar, H Bobby; Thrasher, Adrian J; Chester, Kerry A; Amrolia, Persis J.

Mol Ther ; 2024 Mar 27.

Artículo en Inglés | MEDLINE | ID: mdl-38549377

3.

Hematopoietic stem cell gene editing rescues B-cell development in X-linked agammaglobulinemia.

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.

J Allergy Clin Immunol ; 2024 Mar 11.

Artículo en Inglés | MEDLINE | ID: mdl-38479630

4.

JAK inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID/EBMT-IEWP retrospective study.

Fischer, Marco; Olbrich, Peter; Hadjadj, Jérôme; Aumann, Volker; Bakhtiar, Shahrzad; Barlogis, Vincent; von Bismarck, Philipp; Bloomfield, Markéta; Booth, Claire; Buddingh, Emmeline P; Cagdas, Deniz; Castelle, Martin; Chan, Alice Y; Chandrakasan, Shanmuganathan; Chetty, Kritika; Cougoul, Pierre; Crickx, Etienne; Dara, Jasmeen; Deyà-Martínez, Angela; Farmand, Susan; Formankova, Renata; Gennery, Andrew R; Gonzalez-Granado, Luis Ignacio; Hagin, David; Hanitsch, Leif Gunnar; Hanzlikovà, Jana; Hauck, Fabian; Ivorra-Cortés, José; Kisand, Kai; Kiykim, Ayca; Körholz, Julia; Leahy, Timothy Ronan; van Montfrans, Joris; Nademi, Zohreh; Nelken, Brigitte; Parikh, Suhag; Plado, Silvi; Ramakers, Jan; Redlich, Antje; Rieux-Laucat, Frédéric; Rivière, Jacques G; Rodina, Yulia; Júnior, Pérsio Roxo; Salou, Sarah; Schuetz, Catharina; Shcherbina, Anna; Slatter, Mary A; Touzot, Fabien; Unal, Ekrem; Lankester, Arjan C.

J Allergy Clin Immunol ; 153(1): 275-286.e18, 2024 01.

Artículo en Inglés | MEDLINE | ID: mdl-37935260

5.

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency.

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.

N Engl J Med ; 384(21): 2002-2013, 2021 05 27.

Artículo en Inglés | MEDLINE | ID: mdl-33974366

6.

Quercetin ameliorates XIAP deficiency-associated hyperinflammation.

Chiang, Samuel C C; Owsley, Erika; Panchal, Neelam; Chaturvedi, Vijaya; Terrell, Catherine E; Jordan, Michael B; Mehta, Parinda A; Davies, Stella M; Akeno, Nagako; Booth, Claire; Marsh, Rebecca A.

Blood ; 140(7): 706-715, 2022 08 18.

Artículo en Inglés | MEDLINE | ID: mdl-35687753

7.

Replication competent retrovirus testing (RCR) in the National Gene Vector Biorepository: No evidence of RCR in 1,595 post-treatment peripheral blood samples obtained from 60 clinical trials.

Cornetta, Kenneth; Yao, Jing; House, Kimberley; Duffy, Lisa; Adusumilli, Prasad S; Beyer, Rachel; Booth, Claire; Brenner, Malcolm; Curran, Kevin; Grilley, Bambi; Heslop, Helen; Hinrichs, Christian S; Kaplan, Rosandra N; Kiem, Hans-Peter; Kochenderfer, James; Kohn, Donald B; Mailankody, Sham; Norberg, Scott M; O'Cearbhaill, Roisin E; Pappas, Jennifer; Park, Jae; Ramos, Carlos; Ribas, Antonio; Rivière, Isabelle; Rosenberg, Steven A; Sauter, Craig; Shah, Nirali N; Slovin, Susan F; Thrasher, Adrian; Williams, David A; Lin, Tsai-Yu.

Mol Ther ; 31(3): 801-809, 2023 03 01.

Artículo en Inglés | MEDLINE | ID: mdl-36518078

8.

Lentiviral Gene Transfer Corrects Immune Abnormalities in XIAP Deficiency.

Topal, Joseph; Panchal, Neelam; Barroeta, Amairelys; Roppelt, Anna; Mudde, Annelotte; Gaspar, H Bobby; Thrasher, Adrian J; Houghton, Benjamin C; Booth, Claire.

J Clin Immunol ; 43(2): 440-451, 2023 02.

Artículo en Inglés | MEDLINE | ID: mdl-36329240

9.

Non-osteopenic Bone Pathology After Allo-hematopoietic Stem Cell Transplantation in Patients with Inborn Errors of Immunity.

Golwala, Zainab M; Bhat, Nikita Gireesh; Xu-Bayford, Jinhua; Stankova, Tanja; Adams, Stuart; Morris, Emma C; Qasim, Waseem; Booth, Claire; Worth, Austen; Kusters, Maaike A; Elfeky, Reem.

J Clin Immunol ; 43(5): 1019-1031, 2023 07.

Artículo en Inglés | MEDLINE | ID: mdl-36930409

10.

Absent B cells, agammaglobulinemia, and hypertrophic cardiomyopathy in folliculin-interacting protein 1 deficiency.

Saettini, Francesco; Poli, Cecilia; Vengoechea, Jaime; Bonanomi, Sonia; Orellana, Julio C; Fazio, Grazia; Rodriguez, Fred H; Noguera, Loreani P; Booth, Claire; Jarur-Chamy, Valentina; Shams, Marissa; Iascone, Maria; Vukic, Maja; Gasperini, Serena; Quadri, Manuel; Barroeta Seijas, Amairelys; Rivers, Elizabeth; Mauri, Mario; Badolato, Raffaele; Cazzaniga, Gianni; Bugarin, Cristina; Gaipa, Giuseppe; Kroes, Wilma G M; Moratto, Daniele; van Oostaijen-Ten Dam, Monique M; Baas, Frank; van der Maarel, Silvère; Piazza, Rocco; Coban-Akdemir, Zeynep H; Lupski, James R; Yuan, Bo; Chinn, Ivan K; Daxinger, Lucia; Biondi, Andrea.

Blood ; 137(4): 493-499, 2021 01 28.

Artículo en Inglés | MEDLINE | ID: mdl-32905580

11.

Allosteric inhibition of SHP2 rescues functional T-cell abnormalities in SAP deficiency.

Panchal, Neelam; Houghton, Benjamin Christopher; Vassalou, Elina; Thrasher, Adrian J; Booth, Claire.

J Allergy Clin Immunol ; 150(6): 1507-1516.e7, 2022 12.

Artículo en Inglés | MEDLINE | ID: mdl-35839843

12.

Phenotype, genotype, treatment, and survival outcomes in patients with X-linked inhibitor of apoptosis deficiency.

Yang, Linlin; Booth, Claire; Speckmann, Carsten; Seidel, Markus G; Worth, Austen J J; Kindle, Gerhard; Lankester, Arjan C; Grimbacher, Bodo; Gennery, Andrew R; Seppanen, Mikko R J; Morris, Emma C; Burns, Siobhan O.

J Allergy Clin Immunol ; 150(2): 456-466, 2022 08.

Artículo en Inglés | MEDLINE | ID: mdl-34920033

13.

Preemptive hematopoietic cell transplantation for asymptomatic patients with X-linked lymphoproliferative syndrome type 1.

Tomomasa, Dan; Booth, Claire; Bleesing, Jack J; Isoda, Takeshi; Kobayashi, Chie; Koike, Kazutoshi; Taketani, Takeshi; Sawada, Akihisa; Tamura, Akihiro; Marsh, Rebecca A; Morio, Tomohiro; Gennery, Andrew R; Kanegane, Hirokazu.

Clin Immunol ; 237: 108993, 2022 04.

Artículo en Inglés | MEDLINE | ID: mdl-35367395

14.

Patients with XLP type 1 have variable numbers of NKT cells.

Ralph, Elizabeth; Evans, Josie; Booth, Claire; Gilmour, Kimberly.

Br J Haematol ; 198(1): 151-154, 2022 07.

Artículo en Inglés | MEDLINE | ID: mdl-35355252

15.

Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience.

Kreins, Alexandra Y; Velasco, Helena F; Cheong, Kai-Ning; Rao, Kanchan; Veys, Paul; Worth, Austen; Gaspar, H Bobby; Booth, Claire.

J Clin Immunol ; 42(1): 94-107, 2022 01.

Artículo en Inglés | MEDLINE | ID: mdl-34654999

16.

Reduced-Intensity/Reduced-Toxicity Conditioning Approaches Are Tolerated in XIAP Deficiency but Patients Fare Poorly with Acute GVHD.

Arnold, Danielle E; Nofal, Rofida; Wakefield, Connor; Lehmberg, Kai; Wustrau, Katharina; Albert, Michael H; Morris, Emma C; Heimall, Jennifer R; Bunin, Nancy J; Kumar, Ashish; Jordan, Michael B; Cole, Theresa; Choo, Sharon; Brettig, Tim; Speckmann, Carsten; Ehl, Stephan; Salamonowicz, Malgorzata; Wahlstrom, Justin; Rao, Kanchan; Booth, Claire; Worth, Austen; Marsh, Rebecca A.

J Clin Immunol ; 42(1): 36-45, 2022 01.

Artículo en Inglés | MEDLINE | ID: mdl-34586554

17.

Retrospective, Landmark Analysis of Long-term Adult Morbidity Following Allogeneic HSCT for Inborn Errors of Immunity in Infancy and Childhood.

Day, James W; Elfeky, Reem; Nicholson, Bethany; Goodman, Rupert; Pearce, Rachel; Fox, Thomas A; Worth, Austen; Booth, Claire; Veys, Paul; Carpenter, Ben; Hough, Rachael; Gaspar, H Bobby; Titman, Penny; Ridout, Deborah; Workman, Sarita; Hernandes, Fernando; Sandford, Kit; Laurence, Arian; Campbell, Mari; Burns, Siobhan O; Morris, Emma C.

J Clin Immunol ; 42(6): 1230-1243, 2022 08.

Artículo en Inglés | MEDLINE | ID: mdl-35579633

18.

Gene therapy for primary immunodeficiency.

Booth, Claire; Romano, Rosa; Roncarolo, Maria Grazia; Thrasher, Adrian J.

Hum Mol Genet ; 28(R1): R15-R23, 2019 10 01.

Artículo en Inglés | MEDLINE | ID: mdl-31297531

19.

Gene therapy for primary immunodeficiencies.

Fox, Thomas A; Booth, Claire.

Br J Haematol ; 193(6): 1044-1059, 2021 06.

Artículo en Inglés | MEDLINE | ID: mdl-33336808

20.

T cell gene therapy to treat immunodeficiency.

Panchal, Neelam; Ghosh, Sujal; Booth, Claire.

Br J Haematol ; 192(3): 433-443, 2021 02.

Artículo en Inglés | MEDLINE | ID: mdl-33280098

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