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Journal Articles

Phase II Study of post transplant cyclophosphamide-based graft versus host disease prophylaxis after HLA-mismatched unrelated donor transplantation and reduced intensity conditioning: Results from the NMDP-sponsored access expansion Study

Free
Mahasweta Gooptu, Monzr M. Al Malki, Stephanie Bo-Subait, Brent Logan, Erin Leckrone, Sarah Smith, Heather Stefanski, Jeffery Auletta, Stephen Spellman, Craig Malmberg, Medhat Askar, Rachel Cusatis, Farhad Khimani, Dipenkumar Modi, Mehdi Hamadani, Martin Maiers, Joseph Stanek, Javier Meade, Uttam Rao, Jordan Milner, Ramzi Abboud, Katarzyna Jamieson, George Carrum, Bhagirathbhai Dholaria, William Hogan, Ran Reshef, Satyajit Kosuri, Rachel Cook, Karen Ballen, Alison Loren, Karilyn Larkin, Sally Arai, Muna Qayed, Sung Choi, Larisa Broglie, Bronwen Shaw, Steven Devine, Antonio Jimenez Jimenez, Brian Shaffer
Journal: Blood
Blood (2025) 146 (Supplement 1): 4232.
https://doi.org/10.1182/blood-2025-4232
Published: 2025
Journal Articles

Mismatching of unrelated donors beyond a single HLA-locus does not adversely impact outcomes at one year following transplantation: Results from the NMDP sponsored ACCESS study

Free
Monzr M. Al Malki, Stephanie Bo-Subait, Brent Logan, Sarah Smith, Erin Leckrone, Heather Stefanski, Jeffery Auletta, Stephen Spellman, Craig Malmberg, Medhat Askar, Rachel Cusatis, Brian Shaffer, Dipenkumar Modi, Farhad Khimani, Mahasweta Gooptu, Mehdi Hamadani, Martin Maiers, Joseph Stanek, Javier Meade, Uttam Rao, Jordan Milner, Ramzi Abboud, Katarzyna Jamieson, George Carrum, Bhagirathbhai Dholaria, William Hogan, Ran Reshef, Satyajit Kosuri, Rachel Cook, Karen Ballen, Alison Loren, Karilyn Larkin, Sally Arai, Muna Qayed, Sung Choi, Larisa Broglie, Bronwen Shaw, Steven Devine, Antonio Jimenez Jimenez
Journal: Blood
Blood (2025) 146 (Supplement 1): 936.
https://doi.org/10.1182/blood-2025-936
Published: 2025
Journal Articles

Living the recovery: One-year qol outcomes for patients receiving ptcy gvhd prophylaxis on the access clinical trial

Free
Rachel Cusatis, Jianqun Kou, Juan (Maggie) Wu, Caitrin Bupp, Deborah Mattila, Sarah Smith, Kathryn Flynn, Jeffery Auletta, Steven Devine, Brent Logan, Bronwen Shaw
Journal: Blood
Blood (2025) 146 (Supplement 1): 6042.
https://doi.org/10.1182/blood-2025-6042
Published: 2025
Journal Articles

ACCELERATE: A platform protocol to investigate post-transplant cyclophosphamide-based graft-versus-host disease prophylaxis in patients with hematologic malignancies undergoing mismatched unrelated donor peripheral blood stem cell transplantation

Free
Sarah Smith, Heather Stefanski, Stephen Spellman, Joseph Stanek, Dillon Corrigan, Brent Logan, Bronwen Shaw, Rachel Cusatis, Erin Leckrone, Jeffery Auletta, Steven Devine
Journal: Blood
Blood (2025) 146 (Supplement 1): 6035.
https://doi.org/10.1182/blood-2025-6035
Published: 2025
Journal Articles

Description of an Adolescent and Young Adult Sickle Cell Transition Program: Using Clinical Practices to Inform Clinic Development

Free
Sarah Smith, Jessica O'Brien, Barbara Mclain, Suzanne Braniecki, Edo Schaefer, Lila Pereira
Journal: Blood
Blood (2024) 144 (Supplement 1): 7537.
https://doi.org/10.1182/blood-2024-211923
Published: 2024
Journal Articles

Characterization of Multicellular Niches Supporting Hematopoietic Stem Cells within Distinct Zones

Free
Ruochen Dong, Hua Li, Xi He, Chen Wang, Anoja Perera, Seth Malloy, Jonathon Russell, Wenting Li, Kaitlyn Petentler, Xinjian Mao, Zhe Yang, Michael Epp, Kate Hall, Allison Scott, Mary C McKinney, Shengping Huang, Sarah Smith, Mark Hembree, Yongfu Wang, Zulin Yu, Jeff Haug, Jay Unruh, Brian Slaughter, Xunlei Kang, Linheng Li
Journal: Blood
Blood (2024) 144 (Supplement 1): 1306.
https://doi.org/10.1182/blood-2024-208450
Published: 2024
Journal Articles

Addressing Patient Representation in Hematopoietic Cell Transplant Clinical Trials: Insights Gained through the Access Trial

Free
Jeffery J. Auletta, Stephanie Bo-Subait, Abeer Madbouly, Jaime M. Preussler, Janelle Olson, Lindsay Bankole, Sarah Smith, Martin J. Maiers, Yung-Tsi Bolon, Jennifer Novakovich, Kelly Buck, Craig Malmberg, Kim Wadsworth, Stephanie Fingerson, Christa L Meyer, Anna M. DeSalvo, Katie Schoeppner, Jackie Foster, Ben Tweeten, Rachel Cusatis, Deborah Mattila, Caitrin Bupp, Juan Wu, Larisa Broglie, Brent R. Logan, Bronwen E. Shaw, Heather E. Stefanski, Stephen R. Spellman, Steven M. Devine
Journal: Blood
Blood (2024) 144 (Supplement 1): 3672.
https://doi.org/10.1182/blood-2024-198523
Published: 2024
Journal Articles

Non-Genotoxic Conditioning for Hematopoietic Stem Cell Transplant through Engineered Stem Cell Antibody Paired Evasion (ESCAPE)

Free
Nandini Mondal, Alexander Harmon, Elizabeth Budak, Kathy Zhang, Jeffrey Wong, Archita Venugopal Menon, Adam Wolin, Moriah White, Faith Musenge, Adam Camblin, Brent Coisman, Corrina Lucini, Tao Bai, Kangjian Qiao, Wayne Austin, Michelle DeLelys, Hugh Kromer, Lisa Hardy, Megan Law, Raffi Manoukian, Rachel Goldsmith, Kiera Fallon, Mudra Patel, Antoinette Cozier, Colin Lazzara, Jeremy Decker, Rebecca Jenkins, Tanggis Bohnuud, Patricia Feliciano, Seung-Joo Lee, Paul Kopesky, Sarah Smith, Charlotte McDonagh, Nicole Gaudelli, S. Haihua Chu, Adam Hartigan, Gopi Shankar, Giuseppe Ciaramella
Journal: Blood
Blood (2023) 142 (Supplement 1): 7128.
https://doi.org/10.1182/blood-2023-182077
Published: 2023
Journal Articles

Spatial Transcriptomics Reveals Distinct Hematopoietic Stem Cell Niches in Mouse Fetal Liver

Free
Ruochen Dong, Hua Li, Xi He, Anoja Perera, Seth Malloy, Jonathon Russell, Wenting Li, Kaitlyn Petentler, Xinjian Mao, Zhe Yang, Michael Epp, Kate Hall, Allison Scott, Sarah Smith, Mark Hembree, Yongfu Wang, Sean McKinney, Jeff Haug, Jay Unruh, Brian Slaughter, Linheng Li
Journal: Blood
Blood (2023) 142 (Supplement 1): 2700.
https://doi.org/10.1182/blood-2023-189880
Published: 2023
Journal Articles

Engineered Stem Cell Antibody Paired Evasion 1 (ESCAPE-1): Paired HSC Epitope Engineering and Upregulation of Fetal Hemoglobin for Antibody-Mediated Autologous Hematopoietic Stem Cell Therapy Conditioning for the Treatment of Hemoglobinopathies

Free
Nandini Mondal, Alexander Harmon, Elizabeth Budak, Kathy Zhang, Jeffrey Wong, Conrad Rinaldi, Jenny Olins, Kara Hoar, Archita Venugopal Menon, Moriah White, Faith Musenge, Adam Camblin, Adam Wolin, Brent Coisman, Tao Bai, Kangjian Qiao, Mudra Patel, Jeremy Decker, Bob Gantzer, Thomas Leete, Yi Yu, Tanggis Bohnuud, Seung-Joo Lee, Sarah Smith, Charlotte F McDonagh, Nicole M. Gaudelli, S. Haihua Chu, Adam J. Hartigan, Giuseppe Ciaramella
Journal: Blood
Blood (2022) 140 (Supplement 1): 4487–4488.
https://doi.org/10.1182/blood-2022-169120
Published: 2022
Journal Articles

Conversion of HbS to Hb G-Makassar By Adenine Base Editing Is Compatible with Normal Hemoglobin Function

Free
S. Haihua Chu, Manuel Ortega, Paty Feliciano, Valerie Winton, Chavonna Xu, Daniel Haupt, Teresa McDonald, Salette Martinez, Alexander Liquori, Jeffrey Marshall, Daisy Lam, Jenny Olins, Conrad Rinaldi, Kyle Rehberger, Colin Lazarra, Jeremy Decker, Bob Gantzer, Tanggis Bohnuud, David Born, Luis Barrera, Bo Yan, Ian Slaymaker, Michael S. Packer, Sarah Smith, Carlo Zambonelli, Matt Lee, Nicole M. Gaudelli, Adam J. Hartigan, Giuseppe Ciaramella
Journal: Blood
Blood (2021) 138 (Supplement 1): 951.
https://doi.org/10.1182/blood-2021-150922
Published: 2021
Journal Articles

Indirect Treatment Comparisons of the Effect of Fedratinib Versus Navitoclax Plus Ruxolitinib on Spleen Volume and Symptoms in Patients with Myelofibrosis and Prior Ruxolitinib Treatment

Free
Pranav Abraham, Xiaomeng Liao, Manoj Chevli, Sarah Smith
Journal: Blood
Blood (2021) 138 (Supplement 1): 4626.
https://doi.org/10.1182/blood-2021-145862
Published: 2021
Journal Articles

Complementary Base Editing Approaches for the Treatment of Sickle Cell Disease and Beta Thalassemia

Free
Ling Lin, PhD, Adrian P. Rybak, PhD, Conrad Rinaldi, M.S., Jonathan Yen, PhD, Yanfang Fu, PhD, Elsie Akrawi, Sarah Smith, Scott J. Haskett, M.S., Minerva E. Sanchez, Yeh-Chuin Poh, PhD, Michael S. Packer, PhD, Nicole M. Gaudelli, PhD, Manmohan Singh, PhD, Giuseppe Ciaramella, PhD, B.Sc., Dana N. Levasseur, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3352.
https://doi.org/10.1182/blood-2019-126710
Published: 2019
Journal Articles

Highly Efficient Multiplexed Base Editing with Minimized Off-Targets for the Development of Universal CAR-T Cells to Treat Pediatric T-ALL

Free
Jason M Gehrke, PhD, Aaron Edwards, MS, Ryan C. Murray, MS, Amy Shaw, MS, Yeh-Chuin Poh, PhD, Sarah Smith, Yi Yu, PhD, Nicole M. Gaudelli, PhD, Waseem Qasim, MBBS, PhD, Giuseppe Ciaramella, PhD, B.Sc.
Journal: Blood
Blood (2019) 134 (Supplement_1): 5127.
https://doi.org/10.1182/blood-2019-130562
Published: 2019
Journal Articles

Clonal Analysis of Human Bone Marrow CD34+ Cells Edited By BCL11A-Targeting Zinc Finger Nucleases Reveals Clinically Relevant Levels of Fetal Globin Expression in Edited Erythroid Progeny

Free
Kai-Hsin Chang, PhD, Timothy Sullivan, MS, Mei Liu, BS, Xiao Yang, PhD, Chao Sun, PhD, Benjamin Vieira, BS, Ming Zhang, PhD, Vu P. Hong, PhD, Kai Chen, MS, Sarah Smith, BS, Siyuan Tan, PhD, Andreas Reik, PhD, Fyodor D Urnov, PhD, Edward J. Rebar, PhD, Olivier Danos, PhD, Haiyan Jiang, PhD
Journal: Blood
Blood (2015) 126 (23): 3234.
https://doi.org/10.1182/blood.V126.23.3234.3234
Published: 2015
Journal Articles

Genome Editing of the Bcl11A Erythroid Specific Enhancer in Bone Marrow Derived Hematopoietic Stem and Progenitor Cells for the Treatment of Sickle Cell Disease

Free
Siyuan Tan, PhD, Kai-Hsin Chang, PhD, Sarah Smith, BS, Kai Chen, MS, Timonthy Sullivan, BS, Qianhe Zhou, PhD, Andreas Reik, PhD, Fyodor D Urnov, PhD, Edward J. Rebar, PhD, Olivier Danos, PhD, Haiyan Jiang, PhD
Journal: Blood
Blood (2015) 126 (23): 203.
https://doi.org/10.1182/blood.V126.23.203.203
Published: 2015
Journal Articles

Enhancing the Acute Hemostatic Efficacy in Cynomolgus Monkeys By Targeting Activated Coagulation Factor VII to Platelets

Free
Siyuan Tan, PhD, Joe Salas, PhD, Kai Chen, Tamera Ashworth, Sarah Smith, Elena Kistanova, Nancy Moore, Marisol Acosta, Anxhela Kole, David R. Light, PhD, Robert Peters, PhD, Haiyan Jiang, PhD
Journal: Blood
Blood (2014) 124 (21): 1488.
https://doi.org/10.1182/blood.V124.21.1488.1488
Published: 2014