Gene Therapies for Primary Immune Deficiencies

by Lisa A. Kohn, Donald B. Kohn
Published 2021-02-01

PGE2 and Poloxamer Synperonic F108 Enhance Transduction of Human HSPCs with a β-Globin Lentiviral Vector

by Katelyn E. Masiuk, Ruixue Zhang, Kyle Osborne, Roger P. Hollis, Beatriz Campo-Fernandez, Donald B. Kohn
Published 2019-06-01

Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

by Denise A. Carbonaro-Sarracino, Krista Chun, Danielle N. Clark, Michael L. Kaufman, Xiangyang Jin, Xiaoyan Wang, Donald B. Kohn
Published 2021-03-01

Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease

by Valentina Poletti, Fabrizia Urbinati, Sabine Charrier, Guillaume Corre, Roger P. Hollis, Beatriz Campo Fernandez, Samia Martin, Michael Rothe, Axel Schambach, Donald B. Kohn, Fulvio Mavilio
Published 2018-12-01

Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases

by Carmen F Bjurström, Michelle Mojadidi, John Phillips, Caroline Kuo, Stephen Lai, Georgia R Lill, Aaron Cooper, Michael Kaufman, Fabrizia Urbinati, Xiaoyan Wang, Roger P Hollis, Donald B Kohn
Published 2016-01-01

Domain-swapped T cell receptors improve the safety of TCR gene therapy

by Michael T Bethune, Marvin H Gee, Mario Bunse, Mark S Lee, Eric H Gschweng, Meghana S Pagadala, Jing Zhou, Donghui Cheng, James R Heath, Donald B Kohn, Michael S Kuhns, Wolfgang Uckert, David Baltimore
Published 2016-11-01

Human CLEC9A antibodies deliver NY-ESO-1 antigen to CD141+ dendritic cells to activate naïve and memory NY-ESO-1-specific CD8+ T cells

by Oscar L Haigh, Kirsteen M Tullett, Carina Walpole, Jonathon Cebon, Christopher Schmidt, Liam O'Brien, Nikita Rosendahl, Ghazal Daraj, Irina Caminschi, Eric H Gschweng, Roger P Hollis, Donald B Kohn, Mireille H Lahoud
Published 2020-07-01

The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells

by Zulema Romero, Beatriz Campo-Fernandez, Jennifer Wherley, Michael L Kaufman, Fabrizia Urbinati, Aaron R Cooper, Megan D Hoban, Kismet M Baldwin, Dianne Lumaquin, Xiaoyan Wang, Shantha Senadheera, Roger P Hollis, Donald B Kohn
Published 2015-01-01

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

by Denise A. Carbonaro-Sarracino, Alice F. Tarantal, C. Chang I. Lee, Michael L. Kaufman, Stephen Wandro, Xiangyang Jin, Michele Martinez, Danielle N. Clark, Krista Chun, Colin Koziol, Cinnamon L. Hardee, Xiaoyan Wang, Donald B. Kohn
Published 2020-03-01

Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences

by Richard A. Morgan, Feiyang Ma, Mildred J. Unti, Devin Brown, Paul George Ayoub, Curtis Tam, Lindsay Lathrop, Bamidele Aleshe, Ryo Kurita, Yukio Nakamura, Shantha Senadheera, Ryan L. Wong, Roger P. Hollis, Matteo Pellegrini, Donald B. Kohn
Published 2020-06-01

Global and Local Manipulation of DNA Repair Mechanisms to Alter Site-Specific Gene Editing Outcomes in Hematopoietic Stem Cells

by Elizabeth K. Benitez, Anastasia Lomova Kaufman, Lilibeth Cervantes, Danielle N. Clark, Paul G. Ayoub, Shantha Senadheera, Kyle Osborne, Julie M. Sanchez, Ralph Valentine Crisostomo, Xiaoyan Wang, Nina Reuven, Yosef Shaul, Roger P. Hollis, Zulema Romero, Donald B. Kohn
Published 2020-12-01

Correction to: Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization

by Roni A. Hazim, Saravanan Karumbayaram, Mei Jiang, Anupama Dimashkie, Vanda S. Lopes, Douran Li, Barry L. Burgess, Preethi Vijayaraj, Jackelyn A. Alva-Ornelas, Jerome A. Zack, Donald B. Kohn, Brigitte N. Gomperts, April D. Pyle, William E. Lowry, David S. Williams
Published 2019-02-01

Differentiation of RPE cells from integration-free iPS cells and their cell biological characterization

by Roni A. Hazim, Saravanan Karumbayaram, Mei Jiang, Anupama Dimashkie, Vanda S. Lopes, Douran Li, Barry L. Burgess, Preethi Vijayaraj, Jackelyn A. Alva-Ornelas, Jerome A. Zack, Donald B. Kohn, Brigitte N. Gomperts, April D. Pyle, William E. Lowry, David S. Williams
Published 2017-10-01

Site-Specific Gene Editing of Human Hematopoietic Stem Cells for X-Linked Hyper-IgM Syndrome

by Caroline Y. Kuo, Joseph D. Long, Beatriz Campo-Fernandez, Satiro de Oliveira, Aaron R. Cooper, Zulema Romero, Megan D. Hoban, Alok V. Joglekar, Georgia R. Lill, Michael L. Kaufman, Sorel Fitz-Gibbon, Xiaoyan Wang, Roger P. Hollis, Donald B. Kohn
Published 2018-05-01

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

Antiviral drug screen identifies DNA-damage response inhibitor as potent blocker of SARS-CoV-2 replication

by Gustavo Garcia, Jr., Arun Sharma, Arunachalam Ramaiah, Chandani Sen, Arunima Purkayastha, Donald B. Kohn, Mark S. Parcells, Sebastian Beck, Heeyoung Kim, Malina A. Bakowski, Melanie G. Kirkpatrick, Laura Riva, Karen C. Wolff, Brandon Han, Constance Yuen, David Ulmert, Prabhat K. Purbey, Phillip Scumpia, Nathan Beutler, Thomas F. Rogers, Arnab K. Chatterjee, Gülsah Gabriel, Ralf Bartenschlager, Brigitte Gomperts, Clive N. Svendsen, Ulrich A.K. Betz, Robert D. Damoiseaux, Vaithilingaraja Arumugaswami
Published 2021-04-01

Evidence generation and reproducibility in cell and gene therapy research: A call to action

by Mohamed Abou-el-Enein, Aris Angelis, Frederick R. Appelbaum, Nancy C. Andrews, Susan E. Bates, Arlene S. Bierman, Malcolm K. Brenner, Marina Cavazzana, Michael A. Caligiuri, Hans Clevers, Emer Cooke, George Q. Daley, Victor J. Dzau, Lee M. Ellis, Harvey V. Fineberg, Lawrence S.B. Goldstein, Stephen Gottschalk, Margaret A. Hamburg, Donald E. Ingber, Donald B. Kohn, Adrian R. Krainer, Marcela V. Maus, Peter Marks, Christine L. Mummery, Roderic I. Pettigrew, Joni L. Rutter, Sarah A. Teichmann, Andre Terzic, Fyodor D. Urnov, David A. Williams, Jedd D. Wolchok, Mark Lawler, Cameron J. Turtle, Gerhard Bauer, John P.A. Ioannidis
Published 2021-09-01

Hematopoietic Cell Transplantation in Patients With Primary Immune Regulatory Disorders (PIRD): A Primary Immune Deficiency Treatment Consortium (PIDTC) Survey

by Alice Y. Chan, Jennifer W. Leiding, Xuerong Liu, Brent R. Logan, Lauri M. Burroughs, Eric J. Allenspach, Suzanne Skoda-Smith, Gulbu Uzel, Luigi D. Notarangelo, Mary Slatter, Andrew R. Gennery, Angela R. Smith, Sung-Yun Pai, Sung-Yun Pai, Sung-Yun Pai, Michael B. Jordan, Rebecca A. Marsh, Morton J. Cowan, Christopher C. Dvorak, John A. Craddock, Susan E. Prockop, Shanmuganathan Chandrakasan, Neena Kapoor, Rebecca H. Buckley, Suhag Parikh, Deepak Chellapandian, Benjamin R. Oshrine, Jeffrey J. Bednarski, Megan A. Cooper, Shalini Shenoy, Blachy J. Davila Saldana, Lisa R. Forbes, Caridad Martinez, Elie Haddad, David C. Shyr, Karin Chen, Kathleen E. Sullivan, Jennifer Heimall, Nicola Wright, Monica Bhatia, Geoffrey D. E. Cuvelier, Frederick D. Goldman, Isabelle Meyts, Isabelle Meyts, Holly K. Miller, Markus G. Seidel, Mark T. Vander Lugt, Rosa Bacchetta, Katja G. Weinacht, Jeffrey R. Andolina, Emi Caywood, Hey Chong, Maria Teresa de la Morena, Victor M. Aquino, Evan Shereck, Jolan E. Walter, Jolan E. Walter, Jolan E. Walter, Morna J. Dorsey, Christine M. Seroogy, Linda M. Griffith, Donald B. Kohn, Jennifer M. Puck, Michael A. Pulsipher, Troy R. Torgerson
Published 2020-02-01