An Update from the United States National Heart, Lung, and Blood Institute‐funded Production Assistance for Cellular Therapies (PACT) Program: A Decade of Cell Therapy

by Deborah Wood, Robin Wesselschmidt, Peiman Hematti, Adrian P. Gee, Cliona Rooney, Leslie Silberstein, Myriam Armant, Larry Couture, John E. Wagner, David H. McKenna Jr., Derek Hei, Traci Heath Mondoro, Lisbeth Welniak, Robert Lindblad
Published 2014-04-01

Pre-clinical Safety and Efficacy of Lentiviral Vector-Mediated Ex Vivo Stem Cell Gene Therapy for the Treatment of Mucopolysaccharidosis IIIA

by Stuart M. Ellison, Aiyin Liao, Shaun Wood, Jessica Taylor, Amir Saam Youshani, Sam Rowlston, Helen Parker, Myriam Armant, Alessandra Biffi, Lucas Chan, Farzin Farzaneh, Rob Wynn, Simon A. Jones, Paul Heal, H. Bobby Gaspar, Brian W. Bigger
Published 2019-06-01

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy

by Christian Brendel, Olivier Negre, Michael Rothe, Swaroopa Guda, Geoff Parsons, Chad Harris, Meaghan McGuinness, Daniela Abriss, Alla Tsytsykova, Denise Klatt, Martin Bentler, Danilo Pellin, Lauryn Christiansen, Axel Schambach, John Manis, Helene Trebeden-Negre, Melissa Bonner, Erica Esrick, Gabor Veres, Myriam Armant, David A. Williams
Published 2020-06-01

T cell dynamics and response of the microbiota after gene therapy to treat X-linked severe combined immunodeficiency

by Erik L. Clarke, A. Jesse Connell, Emmanuelle Six, Nadia A. Kadry, Arwa A. Abbas, Young Hwang, John K. Everett, Casey E. Hofstaedter, Rebecca Marsh, Myriam Armant, Judith Kelsen, Luigi D. Notarangelo, Ronald G. Collman, Salima Hacein-Bey-Abina, Donald B. Kohn, Marina Cavazzana, Alain Fischer, David A. Williams, Sung-Yun Pai, Frederic D. Bushman
Published 2018-09-01