Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans

Autor: Natalia Izotova, Christine Rivat, Cristina Baricordi, Elena Blanco, Danilo Pellin, Eleanor Watt, Athina S. Gkazi, Stuart Adams, Kimberly Gilmour, Jinhua Bayford, Claire Booth, H. Bobby Gaspar, Adrian J. Thrasher, Luca Biasco

Publikováno v: Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)

Gene therapy (GT) using haematopoietic stem cells (HSCs) provides an opportunity to trace cell fates in humans, in vivo. Here the authors present evidence in GT patients for a long term lymphoid progenitor population, surviving and maintaining de nov

Externí odkaz: https://doaj.org/article/dced9d3e9d5c441eaba1bc9789351905

Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency

Autor: 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

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

BACKGROUND: Severe combined immunodeficiency due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a rare and life-threatening primary immunodeficiency. METHODS: We treated 50 patients with ADA-SCID (30 in the United States and 20 in the United K

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

Genome-edited, donor-derived allogeneic anti-CD19 chimeric antigen receptor T cells in paediatric and adult B-cell acute lymphoblastic leukaemia: results of two phase 1 studies

Autor: Reuben Benjamin, Charlotte Graham, Deborah Yallop, Agnieszka Jozwik, Oana C Mirci-Danicar, Giovanna Lucchini, Danielle Pinner, Nitin Jain, Hagop Kantarjian, Nicolas Boissel, Marcela V Maus, Matthew J Frigault, André Baruchel, Mohamad Mohty, Athos Gianella-Borradori, Florence Binlich, Svetlana Balandraud, Fabien Vitry, Elisabeth Thomas, Anne Philippe, Sylvain Fouliard, Sandra Dupouy, Ibtissam Marchiq, Maria Almena-Carrasco, Nicolas Ferry, Sylvain Arnould, Cyril Konto, Paul Veys, Waseem Qasim, Antonio Pagliuca, Ghulam Mufti, Piers Patten, Shireen Kassam, Stephen Devereux, Majid Kazmi, Kirsty Cuthill, Victoria Potter, Andrea Kuhnl, Victoria Metaxa, Laarni Bonganay, Orla Stewart, Rose Ellard, Lorraine Catt, Jen Lewis, Farzin Farzaneh, Jackie Chappell, Alice Mason, Vicky Chu, Alan Dunlop, Adeel Saleem, Gary Cheung, Helena Munro, Elka Giemza, Oana Ciocarlie, Jan Chu, Persis Amrolia, Kanchan Rao, Robert Chiesa, Juliana Silva, Annette Hill, Maria Finch, Lindsey Young, Harvinder Hara, Sujith Samarasinghe, Anupama Rao, Ajay Vora, Kimberley Gilmour, Christine Rivat, Clare Murphy, Gulrukh Ahsan, Rasha Said Shamsah, Jesmina James, Sarah Inglott, Gary Wright, Stuart Adams, Natalia Izotova, Marina Konopleva, William Wierda, Elias Jabbour, Partow Kebrieai, Emily Jones, Kara McGee, Marcela Maus, Matthew Frigault, Jami Brown, Vesselina Toncheva, Keagan Casey, Hanno Hock, Meaghan A McKeown, Richard Mathews, Thomas Spitzer, Emmanuel Raffoux, Etienne Lengliné, Raphael Itzykson, Florence Rabian, Jérôme Larghero, Isabelle Madelaine, Elie Azoulay, Emmanuelle Clappier, Sophie Caillat-Zucman, Martine Meunier, Karine Celli-Lebras, Marie-Thérèse Tremorin, Karima Yakouben, Françoise Mechinaud-Heloury, Audrey Grain, Aurélia Alimi, Julie Roupret, Delphine Chaillou, Hélène Cavé, Aurelie Caye-Eude, Odile Fenneteau, Elodie Lainey, Jerome Naudin, Eolia Brissot, Remy Dulery, Florent Malard, Clémence Mediavilla, Agnès Bonnin, Anne Vekhoff, Tounes Ledraa, Anne Daguenel-Nguyen

Publikováno v: The Lancet. 396:1885-1894

Summary Background Genome-edited donor-derived allogeneic anti-CD19 chimeric antigen receptor (CAR) T cells offer a novel form of CAR-T-cell product that is available for immediate clinical use, thereby broadening access and applicability. UCART19 is

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2ec40a8b13cd2746d839da37388e3ee8
https://doi.org/10.1016/s0140-6736(20)32334-5

Long-term lymphoid progenitors independently sustain naïve T and NK cell production in humans

Autor: Eleanor Watt, Stuart Adams, Luca Biasco, Danilo Pellin, Elena Blanco, Athina Soragia Gkazi, H. Bobby Gaspar, Cristina Baricordi, Jinhua Xu Bayford, Christine Rivat, Claire Booth, Kimberly Gilmour, Natalia Izotova, Adrian J. Thrasher

Publikováno v: Nature Communications
Nature Communications, Vol 12, Iss 1, Pp 1-16 (2021)

Our mathematical model of integration site data in clinical gene therapy supported the existence of long-term lymphoid progenitors capable of surviving independently from hematopoietic stem cells. To date, no experimental setting has been available t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e68f61cc17b2c7300e4cdbbd7e0c2e56
https://doi.org/10.1038/s41467-021-21834-9

Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult

Autor: Christine Rivat, Ronjon Chakraverty, Emma C. Morris, Alan D. Salama, Anne Galy, H. Bobby Gaspar, Karen F. Buckland, Adrian J. Thrasher, Ronnie Chee, Rita Tendeiro, Sarita Workman, Thomas A. Fox, Siobhan O. Burns, Frederick D. Bushman, Kimberly Gilmour, Frances Male, Katie Butler, Cecile Duret, Sarah Grace, Katie Snell, Fulvio Mavilio, Hazem Ibrahim, Havinder Hara

Publikováno v: Blood
Blood, 2017, 130 (11), pp.1327-1335. ⟨10.1182/blood-2017-04-777136⟩
Blood, American Society of Hematology, 2017, 130 (11), pp.1327-1335. ⟨10.1182/blood-2017-04-777136⟩

International audience; Until recently, hematopoietic stem cell transplantation was the only curative option for Wiskott-Aldrich syndrome (WAS). The first attempts at gene therapy for WAS using a Upsilon-retroviral vector improved immunological param

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bc04e2ec10605e4bca31f64aac81e7c1
https://doi.org/10.1182/blood-2017-04-777136