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Journal Articles

Epitope Edited Hematopoietic Stem Cells to Enable Synergistic Immunotherapy Combinations for Acute Myeloid Leukemia

Gabriele Casirati, Andrea Cosentino, Adele Mucci, Mohammed S Mahmoud, Iratxe Ugarte Zabala, Jing Zeng, Scott Ficarro, Denise Klatt, Christian Brendel, Alessandro Rambaldi, Jerome Ritz, Jarrod Marto, Danilo Pellin, Daniel E. Bauer, Scott A Armstrong, Pietro Genovese
Journal: Blood
Blood (2023) 142 (Supplement 1): 7130.
https://doi.org/10.1182/blood-2023-189980
Published: 2023
Journal Articles

Outcomes of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome

Clinical Trials & Observations
Roxane Labrosse, Julia I. Chu, Myriam A. Armant, John K. Everett, Danilo Pellin, Niharika Kareddy, Andrew L. Frelinger, Lauren A. Henderson, Amy E. O’Connell, Amlan Biswas, Jet Coenen-van der Spek, Alexandra Miggelbrink, Claudia Fiorini, Hriju Adhikari, Charles C. Berry, Vito Adrian Cantu, Johnson Fong, Jason Jaroslavsky, Derin F. Karadeniz, Quan-Zhen Li, Shantan Reddy, Aoife M. Roche, Chengsong Zhu, Jennifer S. Whangbo, Colleen Dansereau, Brenda Mackinnon, Emily Morris, Stephanie M. Koo, Wendy B. London, Safa Baris, Ahmet Ozen, Elif Karakoc-Aydiner, Jenny M. Despotovic, Lisa R. Forbes Satter, Akihiko Saitoh, Yuta Aizawa, Alejandra King, Mai Anh Thi Nguyen, Vy Do Uyen Vu, Scott B. Snapper, Anne Galy, Luigi D. Notarangelo, Frederic D. Bushman, David A. Williams, Sung-Yun Pai
Journal: Blood
Blood (2023) 142 (15): 1281–1296.
https://doi.org/10.1182/blood.2022019117
Published: 2023
Includes: Supplemental data
Journal Articles

Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial

Erica B. Esrick, Amy Federico, Daniela Abriss, Myriam Armant, Kari Boardman, Christian Brendel, Marioara-Felicia Ciuculescu, Heather Daley, Colleen Dansereau, Augustine Fernandes, Matthew M. Heeney, David G. Justus, Pei-Chi Kao, Annette S. Kim, Donald B. Kohn, Leslie E. Lehmann, Donghui Liu, Wendy B. London, John P Manis, Theodore B. Moore, Emily Morris, Danilo Pellin, Ellen Proeung, Shanna Richard, Gavin D. Roach, Kit L. Shaw, Dayna Terrazas, Shanna L White, David A. Williams
Journal: Blood
Blood (2022) 140 (Supplement 1): 10665–10667.
https://doi.org/10.1182/blood-2022-169089
Published: 2022
Journal Articles

Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models

Jing Zeng, Selami Demirci, My Anh Nguyen, Linda Yingqi Lin, Stacy A. Maitland, Esther Mintzer, Yuxuan Wu, Danilo Pellin, Amornrat Tangprasittipap, Chokdee Vong, Shaina N. Porter, Kevin Luk, Pengpeng Liu, Varun Katta, Marioara-Felicia Ciuculescu, Daniela Abriss, Jonathan Hsu, Naoya Uchida, Khaled Essawi, Robert Donahue, Karl Petri, Vikram Pattanayak, Luca Pinello, Christian Brendel, David A Williams, John P Manis, Shengdar Q. Tsai, Shondra M. Pruett-Miller, J. Keith Joung, Duantida Songdej, Suradej Hongeng, Myriam Armant, Scot A. Wolfe, John F. Tisdale, Daniel E. Bauer
Journal: Blood
Blood (2021) 138 (Supplement 1): 1852.
https://doi.org/10.1182/blood-2021-152634
Published: 2021
Journal Articles

Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans

Natalia Izotova, Christine Rivat, Cristina Baricordi, PhD, Elena Blanco-Alvarez, Danilo Pellin, Eleanor Watt, Soragia Athina Gkazi, PhD, Stuart Adams, PhD, Kimberly Gilmour, Jinhua Xu Bayford, Claire Booth, MBBS, PhD MSc, Bobby H Gaspar, MBBS, PhD BS, Adrian J. Thrasher, MBBS, PhD FMedSci, Luca Biasco, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 50.
https://doi.org/10.1182/blood-2020-137001
Published: 2020
Journal Articles

Clonal Dynamics of Early Responder and Long-Term Surviving CAR-T Cells in Humans

Christine Rivat, Natalia Izotova, Rachel Richardson, MSc, Danilo Pellin, Rachael Hough, Robert Wynn, MD, Kim Champion, Andre Lopes, Sara Ghorashian, FRCPath, PhD, Martin Pule, Adrian J. Thrasher, MBBS, PhD FMedSci, Luca Biasco, PhD, Persis J Amrolia, FRCP, FRCPath, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 52.
https://doi.org/10.1182/blood-2019-125916
Published: 2019
Journal Articles

The Single-Cell Transcriptional Landscape of Human Hematopoietic Progenitors

Mariana Loperfido, PhD, Danilo Pellin, Cristina Baricordi, Samuel Louis Wolock, Allon Moshe Klein, Luca Biasco
Journal: Blood
Blood (2018) 132 (Supplement 1): 1266.
https://doi.org/10.1182/blood-2018-99-116327
Published: 2018
Journal Articles

Comprehensive Clonal Mapping of Hematopoiesis in Vivo in Humans By Retroviral Vector Insertional Barcoding

Luca Biasco, PhD, Serena Scala, Francesca Dionisio, Andrea Calabria, Luca Basso Ricci, Samantha Scaramuzza, Cristina Baricordi, Stefania Giannelli, Victor X Neduva, David J Dow, Danilo Pellin, Paola Vicard, Clelia Di Serio, Eugenio Montini, Luigi Naldini, Alessandro Aiuti
Journal: Blood
Blood (2014) 124 (21): 5.
https://doi.org/10.1182/blood.V124.21.5.5
Published: 2014
Journal Articles

High-definition mapping of retroviral integration sites identifies active regulatory elements in human multipotent hematopoietic progenitors

Claudia Cattoglio, Danilo Pellin, Ermanno Rizzi, Giulietta Maruggi, Giorgio Corti, Francesca Miselli, Daniela Sartori, Alessandro Guffanti, Clelia Di Serio, Alessandro Ambrosi, Gianluca De Bellis, Fulvio Mavilio
Journal: Blood
Blood (2010) 116 (25): 5507–5517.
https://doi.org/10.1182/blood-2010-05-283523
Published: 2010
Includes: Supplemental data
Journal Articles

Uncovering Haematopoietic System Dynamics and Single Multipotent Progenitors Activity In Vivo In Humans by Retroviral Tagging.

Luca Biasco, Cristina Baricordi, Stefania Merella, Cynthia Bartholomae, Alessandro Ambrosi, Danilo Pellin, Clelia Di Serio, Christof Von Kalle, Manfred Schmidt, Alessandro Aiuti
Journal: Blood
Blood (2010) 116 (21): 2611.
https://doi.org/10.1182/blood.V116.21.2611.2611
Published: 2010