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

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

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

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

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