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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

Phase I Study of PHE885, a Fully Human BCMA-Directed CAR-T Cell Therapy for Relapsed/Refractory Multiple Myeloma Manufactured in <2 Days Using the T-Charge TM Platform

Adam S. Sperling, Sarah Nikiforow, Omar Nadeem, Clifton C Mo, Jacob P. Laubach, Kenneth C. Anderson, Alejandro Alonso, Shuntaro Ikegawa, Rao Prabhala, Diego Hernandez Rodriguez, Heather Daley, Kit L. Shaw, Yohei Arihara, Soudeh Ansari, David S Quinn, David Pearson, Anniesha Hack, Louise M Treanor, Dexiu Bu, Jennifer Mataraza, Lawrence Rispoli, Marc Credi, Jerome Ritz, Serena De Vita, Nikhil C. Munshi
Journal: Blood
Blood (2021) 138 (Supplement 1): 3864.
https://doi.org/10.1182/blood-2021-146646
Published: 2021
Journal Articles

Phase 1 Study of CD37-Directed CAR T Cells in Patients with Relapsed or Refractory CD37+ Hematologic Malignancies

Matthew J. Frigault, Yi-Bin Chen, Kathleen M.E. Gallagher, Nora K. Horick, Areej El-Jawahri, Irene Scarfò, Marc Wehrli, Lu Huang, Keagan Casey, Daniella Cook, Thomas Spitzer, Steven McAfee, Frederic I. Preffer, Myriam Armant, Kit L. Shaw, Heather Daley, Sarah Nikiforow, Jerome Ritz, Marcela V. Maus
Journal: Blood
Blood (2021) 138 (Supplement 1): 653.
https://doi.org/10.1182/blood-2021-146236
Published: 2021
Journal Articles

Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency

Clinical Trials & Observations
Bryanna Reinhardt, Omar Habib, Kit L. Shaw, Elizabeth Garabedian, Denise A. Carbonaro-Sarracino, Dayna Terrazas, Beatriz Campo Fernandez, Satiro De Oliveira, Theodore B. Moore, Alan K. Ikeda, Barbara C. Engel, Gregory M. Podsakoff, Roger P. Hollis, Augustine Fernandes, Connie Jackson, Sally Shupien, Suparna Mishra, Alejandra Davila, Jack Mottahedeh, Andrej Vitomirov, Wenzhao Meng, Aaron M. Rosenfeld, Aoife M. Roche, Pascha Hokama, Shantan Reddy, John Everett, Xiaoyan Wang, Eline T. Luning Prak, Kenneth Cornetta, Michael S. Hershfield, Robert Sokolic, Suk See De Ravin, Harry L. Malech, Frederic D. Bushman, Fabio Candotti, Donald B. Kohn
Journal: Blood
Blood (2021) 138 (15): 1304–1316.
https://doi.org/10.1182/blood.2020010260
Published: 2021
Includes: Supplemental data
Journal Articles

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded Cohort

Donald B. Kohn, MD, Kit L. Shaw, PhD, Elizabeth Garabedian, RN, MS, Denise Ann Carbonaro-Sarracino, PhD, Theodore B. Moore, MD, Satiro N. De Oliveira, MD, Gay M. Crooks, MD, John Tse, Sally Shupien, Dayna Terrazas, Alejandra Davila, Amalia Icreverzi, PhD, Allen Yu, Krista M. Chun, Christian E. Casas, Provaboti Barman, PhD, Maritess Coronel, Beatriz Campo Fernandez, PhD, Ruixue Zhang, Roger P. Hollis, PhD, Chilenwa Uzowuru, Hilory Ricketts, Jinhua Xu Bayford, Valentina Trevisan, Serena Arduini, PhD, Frances Lynn, Mahesh Kudari, MBBS, MA, Andrea Spezzi, MD MBBS, Lilith Reeves, MS, MT(ASCP), Kenneth Cornetta, Robert A. Sokolic, MD, Roberta Parrott, BS, Rebecca Buckley, MD, Claire Booth, Fabio Candotti, MD, Harry L. Malech, MD, Adrian J. Thrasher, MBBS, PhD FMedSci, H. Bobby Gaspar, MBBS, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3345.
https://doi.org/10.1182/blood-2019-123432
Published: 2019
Journal Articles

Gene therapy for adenosine deaminase–deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans

Clinical Trials & Observations
Fabio Candotti, Kit L. Shaw, Linda Muul, Denise Carbonaro, Robert Sokolic, Christopher Choi, Shepherd H. Schurman, Elizabeth Garabedian, Chimene Kesserwan, G. Jayashree Jagadeesh, Pei-Yu Fu, Eric Gschweng, Aaron Cooper, John F. Tisdale, Kenneth I. Weinberg, Gay M. Crooks, Neena Kapoor, Ami Shah, Hisham Abdel-Azim, Xiao-Jin Yu, Monika Smogorzewska, Alan S. Wayne, Howard M. Rosenblatt, Carla M. Davis, Celine Hanson, Radha G. Rishi, Xiaoyan Wang, David Gjertson, Otto O. Yang, Arumugam Balamurugan, Gerhard Bauer, Joanna A. Ireland, Barbara C. Engel, Gregory M. Podsakoff, Michael S. Hershfield, R. Michael Blaese, Robertson Parkman, Donald B. Kohn
Journal: Blood
Blood (2012) 120 (18): 3635–3646.
https://doi.org/10.1182/blood-2012-02-400937
Published: 2012
Includes: Supplemental data
Journal Articles

Augmentation of Post-Transplant Immunity: Antigen Encounter at Time of Hematopoietic Stem Cell Transplantation Enhances Antigen-Specific Donor T Cell Responses in the Post-Transplant Repertoire.

Craig A. Mullen, MD PhD, Ulker Kocak, MD, Joanne L. Shaw, BS, Shahram Mori, PhD
Journal: Blood
Blood (2004) 104 (11): 2246.
https://doi.org/10.1182/blood.V104.11.2246.2246
Published: 2004
Journal Articles

Amifostine (WR-2721) shortens the engraftment period of 4- hydroperoxycyclophosphamide-purged bone marrow in breast cancer patients receiving high-dose chemotherapy with autologous bone marrow support

EJ Shpall, SM Stemmer, L Hami, WA Franklin, L Shaw, HS Bonner, SI Bearman, WP Peters, RC Jr Bast, W McCulloch
Journal: Blood
Blood (1994) 83 (11): 3132–3137.
https://doi.org/10.1182/blood.V83.11.3132.3132
Published: 1994