Gene therapy and genome editing for primary immunodeficiency diseases

by Zhi-Yong Zhang, Adrian J. Thrasher, Fang Zhang
Published 2020-03-01

Gene Editing and Genotoxicity: Targeting the Off-Targets

by Georges Blattner, Alessia Cavazza, Adrian J. Thrasher, Giandomenico Turchiano
Published 2020-12-01

Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency

by Claudia A Montiel-Equihua, Adrian J Thrasher, H Bobby Gaspar
Published 2009-12-01

The Wiskott-Aldrich Syndrome: The Actin Cytoskeleton and Immune Cell Function

by Michael P. Blundell, Austen Worth, Gerben Bouma, Adrian J. Thrasher
Published 2010-01-01

Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.

by Sayandip Mukherjee, Giorgia Santilli, Michael P Blundell, Susana Navarro, Juan A Bueren, Adrian J Thrasher
Published 2011-03-01

WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation

by Pilar Muñoz, María Tristán-Manzano, Almudena Sánchez-Gilabert, Giorgia Santilli, Anne Galy, Adrian J. Thrasher, Francisco Martin
Published 2020-12-01

Lentiviral vector transduction of spermatozoa as a tool for the study of early development

by Anil Chandrashekran, Ihsan Isa, Jayesh Dudhia, Adrian J. Thrasher, Nicholas Dibb, Colin Casimir, Carol Readhead, Robert Winston
Published 2014-01-01

Loss of Janus Associated Kinase 1 Alters Urothelial Cell Function and Facilitates the Development of Bladder Cancer

by Vanessa Daza-Cajigal, Vanessa Daza-Cajigal, Vanessa Daza-Cajigal, Vanessa Daza-Cajigal, Vanessa Daza-Cajigal, Adriana S. Albuquerque, Joanna Pearson, Jennifer Hinley, Andrew S. Mason, Jens Stahlschmidt, Jens Stahlschmidt, Adrian J. Thrasher, Adrian J. Thrasher, Vibhash Mishra, Jennifer Southgate, Siobhan O. Burns, Siobhan O. Burns
Published 2019-09-01

Lentiviral Vector Production Titer Is Not Limited in HEK293T by Induced Intracellular Innate Immunity

by Carolina B. Ferreira, Rebecca P. Sumner, Maria T. Rodriguez-Plata, Jane Rasaiyaah, Richard S. Milne, Adrian J. Thrasher, Waseem Qasim, Greg J. Towers
Published 2020-06-01

Wiskott Aldrich syndrome protein regulates non-selective autophagy and mitochondrial homeostasis in human myeloid cells

by Elizabeth Rivers, Rajeev Rai, Jonas Lötscher, Michael Hollinshead, Gasper Markelj, James Thaventhiran, Austen Worth, Alessia Cavazza, Christoph Hess, Mona Bajaj-Elliott, Adrian J Thrasher
Published 2020-11-01

Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency

by Valentina Poletti, Sabine Charrier, Guillaume Corre, Bernard Gjata, Alban Vignaud, Fang Zhang, Michael Rothe, Axel Schambach, H. Bobby Gaspar, Adrian J. Thrasher, Fulvio Mavilio
Published 2018-06-01

Dendritic cell-expressed common gamma-chain recruits IL-15 for trans-presentation at the murine immunological synapse [version 2; referees: 2 approved]

by Chiara Beilin, Kaushik Choudhuri, Gerben Bouma, Dessislava Malinova, Jaime Llodra, David L. Stokes, Motumu Shimaoka, Timothy A. Springer, Michael L. Dustin, Adrian J. Thrasher, Siobhan O. Burns
Published 2018-10-01

Lentiviral vectors can be used for full-length dystrophin gene therapy

by John R. Counsell, Zeinab Asgarian, Jinhong Meng, Veronica Ferrer, Conrad A. Vink, Steven J. Howe, Simon N. Waddington, Adrian J. Thrasher, Francesco Muntoni, Jennifer E. Morgan, Olivier Danos
Published 2017-03-01

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys

by Julien Baruteau, Sharon C. Cunningham, Berna Seker Yilmaz, Dany P. Perocheau, Simon Eaglestone, Derek Burke, Adrian J. Thrasher, Simon N. Waddington, Leszek Lisowski, Ian E. Alexander, Paul Gissen
Published 2021-12-01

The tight junction associated signalling proteins ZO-1 and ZONAB regulate retinal pigment epithelium homeostasis in mice.

by Anastasios Georgiadis, Marion Tschernutter, James W B Bainbridge, Kamaljit S Balaggan, Freya Mowat, Emma L West, Peter M G Munro, Adrian J Thrasher, Karl Matter, Maria S Balda, Robin R Ali
Published 2010-12-01

Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome

by Rajeev Rai, Marianna Romito, Elizabeth Rivers, Giandomenico Turchiano, Georges Blattner, Winston Vetharoy, Dariusz Ladon, Geoffroy Andrieux, Fang Zhang, Marta Zinicola, Diego Leon-Rico, Giorgia Santilli, Adrian J. Thrasher, Alessia Cavazza
Published 2020-08-01

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

by 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
Published 2021-03-01

Biallelic JAK1 mutations in immunodeficient patient with mycobacterial infection

by Davide Eletto, Siobhan O. Burns, Ivan Angulo, Vincent Plagnol, Kimberly C. Gilmour, Frances Henriquez, James Curtis, Miguel Gaspar, Karolin Nowak, Vanessa Daza-Cajigal, Dinakantha Kumararatne, Rainer Doffinger, Adrian J. Thrasher, Sergey Nejentsev
Published 2016-12-01

Wiskott-Aldrich syndrome protein regulates autophagy and inflammasome activity in innate immune cells

by Pamela P. Lee, Damián Lobato-Márquez, Nayani Pramanik, Andrea Sirianni, Vanessa Daza-Cajigal, Elizabeth Rivers, Alessia Cavazza, Gerben Bouma, Dale Moulding, Kjell Hultenby, Lisa S. Westerberg, Michael Hollinshead, Yu-Lung Lau, Siobhan O. Burns, Serge Mostowy, Mona Bajaj-Elliott, Adrian J. Thrasher
Published 2017-11-01

WASP-mediated regulation of anti-inflammatory macrophages is IL-10 dependent and is critical for intestinal homeostasis

by Amlan Biswas, Dror S. Shouval, Alexandra Griffith, Jeremy A. Goettel, Michael Field, Yu Hui Kang, Liza Konnikova, Erin Janssen, Naresh Singh Redhu, Adrian J. Thrasher, Talal Chatila, Vijay K. Kuchroo, Raif S Geha, Luigi D. Notarangelo, Sung-Yun Pai, Bruce H. Horwitz, Scott B. Snapper
Published 2018-05-01