1-20 of 190
gene editing
Close
Save search
Follow your search
Access your saved searches in your account
Would you like to receive an alert when new items match your search?
Close Modal
Sort by
Journal Articles

Introduction to a review series on gene therapy and gene editing for sickle cell disease and hemophilia

Catherine M. Bollard
Journal: Blood
Blood (2021) 138 (11): 913.
https://doi.org/10.1182/blood.2021012891
Published: 2021
Journal Articles

Enhanced homology-directed repair for highly efficient gene editing in hematopoietic stem/progenitor cells

Suk See De Ravin, Julie Brault, Ronald J. Meis, Siyuan Liu, Linhong Li, Mara Pavel-Dinu, Cicera R. Lazzarotto, Taylor Liu, Sherry M. Koontz, Uimook Choi, Colin L. Sweeney, Narda Theobald, GaHyun Lee, Aaron B. Clark, Sandra S. Burkett, Benjamin P. Kleinstiver, Matthew H. Porteus, Shengdar Tsai, Douglas B. Kuhns, Gary A. Dahl, Stephen Headey, Xiaolin Wu, Harry L. Malech
Journal: Blood
Blood (2021) 137 (19): 2598–2608.
https://doi.org/10.1182/blood.2020008503
Published: 2021
Includes: Supplemental data
Journal Articles

Disease Modeling of Severe Congenital Neutropenia Using CRISPR/Cas9 Gene Correction or Knockout of ELANE in Patients Derived Induced Pluripotent Stem Cells

Masoud Nasri, MSc, Benjamin Dannenmann, Perihan Mir, Malte U Ritter, Diana Amend, Yun Xu, Maksim Klimiankou, Cornelia Zeidler, Karl Welte, Julia Skokowa, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 434.
https://doi.org/10.1182/blood-2019-129991
Published: 2019
Journal Articles

Inherited GATA3 Variants Associated with Positive Minimal Residual Disease in Childhood B-ALL Via Autophagy-Induced Asparaginase Resistance

Chunjie Li, PhD, Jiabi Qian, M.Sc, Chuang Jiang, PhD, Ziping LI, B.S, Hui Zhang, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 654.
https://doi.org/10.1182/blood-2019-123575
Published: 2019
Journal Articles

Heterozygous Mutations of Clpb As a Newly Identified and Frequent Cause of Severe Congenital Neutropenia

Julia T Warren, MD PhD, Peeradol Wattanasirakul, David H Spencer, MD, Adam Locke, PhD, Vahagn Makaryan, MD, Audrey Anna Bolyard, RN, BS, Karl Welte, Julia Skokowa, MD PhD, David C. Dale, MD, Daniel C. Link, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 433.
https://doi.org/10.1182/blood-2019-129635
Published: 2019
Journal Articles

CRISPR/Cas9 Mediated ELANE Knock-out Restores Survival and Granulocytic Differentiation of HL60 Cells Expressing Mutant Neutrophil Elastase: Is Neutrophil Elastase a Dispensible Granulocyte Protease?

Vahagn Makaryan, MD, Isabella N. Archibald, Merideth L. Kelley, MS, Breanna Fletcher, BSc, David C. Dale, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 435.
https://doi.org/10.1182/blood-2019-124988
Published: 2019
Journal Articles

Functional Interactions between Transcription Factors Involved in Myeloma Pathogenesis - Biological and Therapeutic Implications

Sondra L. Downey-Kopyscinski, PhD, Ricardo De Matos Simoes, PhD, Ryosuke Shirasaki, MD, Huihui Tang, PhD, Brian Glassner, PhD, Olga Dashevsky, PhD, Minasri Borah, Megan Bariteau, B.Sc., Richard Groen, John G Doench, PhD, Constantine S. Mitsiades, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 315.
https://doi.org/10.1182/blood-2019-127502
Published: 2019
Journal Articles

Functional Characterization of E3 Ligases and Their Regulators: Therapeutic Implications for Development of New Proteolysis-Targeting Chimeric Degraders of Oncoproteins

Ryosuke Shirasaki, MD PhD, Sara Gandolfi, MD, Ricardo De Matos Simoes, PhD, Geoffrey M Matthews, PhD, Dennis Buckley, Joseline Raja, Quinlan L Sievers, PhD, Olga Dashevsky, PhD, Haley Poarch, Huihui Tang, PhD, Megan Bariteau, BSc, Michal Sheffer, PhD, Sondra L. Downey-Kopyscinski, PhD, Paul Hengeveld, Brian Glassner, PhD, Eugen Dhimolea, PhD, Christopher J. Ott, PhD, Tinghu Zhang, PhD, Nicholas P Kwiatkowski, PhD, Jacob P. Laubach, MD, Robert L. Schlossman, MD, Paul G. Richardson, MD, Aedin Culhane, PhD, Richard W.J. Groen, PhD, Eric Fischer, Benjamin L. Ebert, MD PhD, Francisca Vazquez, PhD, Aviad Tsherniak, PhD, William C. Hahn, MD PhD, Joan Levy, PhD, Daniel Auclair, Jonathan D. Licht, MD, Jonathan J Keats, PhD, Lawrence H. Boise, PhD, James Bradner, MD, Nathanael Gray, Constantine S. Mitsiades, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 318.
https://doi.org/10.1182/blood-2019-130549
Published: 2019
Journal Articles

The Inherited Platelet Disorder of RUNX1 Haploinsufficiency (Familial Platelet Disorder with Associated Myeloid Malignancy (FPDMM)) Has a Marked Deficiency of Megakaryocyte-Biased Hematopoietic Progenitor Cells: Mechanistic Studies and Drug Correction

Brian Estevez, PhD, Sara Borst, BS, Danuta Jadwiga Jarocha, PhD, Varun Sudunagunta, Michael Gonzalez, PhD, Peng Gao, Kai Tan, Deborah L French, PhD, Paul Gadue, PhD, Mortimer Poncz, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 220.
https://doi.org/10.1182/blood-2019-124851
Published: 2019
Journal Articles

Statins Enhance Killing of Multiple Myeloma Cells By the BCL-2 Inhibitor Venetoclax and the MCL-1 Inhibitor S63845

David Fruman, PhD, Dennis Juarez, B.S., Grace Diep, Undergraduate, Falisha Nguyen, Undergraduate, Vikas A. Gupta, MD PhD, Lawrence H. Boise, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4413.
https://doi.org/10.1182/blood-2019-131218
Published: 2019
Journal Articles

Systematic Characterization of Genes Representing Preferential Molecular Vulnerabilities for Myeloma Cells Compared to Other Neoplasias - Implications for the Biology and Therapeutic Targeting of Myeloma

Ricardo De Matos Simoes, PhD, Ryosuke Shirasaki, MD PhD, Sondra L. Downey-Kopyscinski, PhD, Geoffrey Matthews, PhD, Hu Yiguo, PhD, Michal Sheffer, PhD, Eugen Dhimolea, PhD, Olga Dashevsky, PhD, Sara Gandolfi, MD, Joshua Dempster, PhD, Robin M. Meyers, PhD, Jordan Bryan, James M. McFarland, Johanna Brueggenthies, Huihui Tang, PhD, Megan Bariteau, BSc, Minasri Borah, Quinlan L Sievers, PhD, Benjamin Ebert, MD, Brian Glassner, PhD, Christopher J. Ott, PhD, Nicholas P Kwiatkowski, PhD, John G Doench, PhD, Daniel Auclair, Joan Levy, PhD, Lawrence H. Boise, PhD, Jonathan J Keats, PhD, Jonathan D. Licht, MD, Richard Groen, Nathanael Gray, Aedin Culhane, PhD, William C. Hahn, MD PhD, Francisca Vazquez, PhD, Aviad Tsherniak, PhD, Constantine S. Mitsiades, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4407.
https://doi.org/10.1182/blood-2019-130901
Published: 2019
Journal Articles

Use of CD70 Targeted Chimeric Antigen Receptor (CAR) T Cells for the Treatment of Acute Myeloid Leukemia (AML)

Mark Leick, MD, Irene Scarfò, PhD, Bryan D. Choi, MD PhD, Rebecca Larson, BS, Amanda A Bouffard, Ana Castano, MD, Maria L. Cabral, BS, Andrea Schmidts, MD, Matthew J. Frigault, MD, Marcela V. Maus, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4443.
https://doi.org/10.1182/blood-2019-127154
Published: 2019
Journal Articles

Manufacture of an Allogeneic CAR-T Stem Cell Memory Product Candidate for Multiple Myeloma, P-Bcma-ALLO1, Is Robust, Reproducible and Highly Scalable

Stacey A Cranert, PhD, Maximilian Richter, PhD, Min Tong, MS, Leslie Weiss, MS, Yening Tan, MS, Eric M. Ostertag, MD PhD, Julia Coronella, PhD, Devon J Shedlock, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 4445.
https://doi.org/10.1182/blood-2019-131839
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

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

CRISPR/dCas9 (D10A) -Mediated Hb CS Gene Editing and Identification of Genetically Modified Fibroblasts

Lian Yu, Wei Hao Wu, WANG Shunqing, Ken H. Young, Funeng Jiang, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 5754.
https://doi.org/10.1182/blood-2019-129931
Published: 2019
Journal Articles

Fanconi Anemia: A Paradigm for Understanding DNA Repair During Replication.

Agata Smogorzewska, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): SCI-32.
https://doi.org/10.1182/blood-2019-121229
Published: 2019
Journal Articles

Targeting Bcl-2 Proteins in Myeloma

Martine Amiot, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): SCI-11.
https://doi.org/10.1182/blood-2019-121103
Published: 2019
Journal Articles

Ex vivo Engineering of Red Blood Cells

Jan Frayne, BsC, PhD, Deborah E Daniels, Jan Frayne, BsC PhD, Timothy J Satchwell, PhD, Joseph Hawksworth, BSc PhD, Belinda K Singleton, Tatyana N Andrienko, PhD, Marieangela C Wilson, Sabine Kupzig, Marjolein Meinders, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Ivan Ferrer Vicens, PhD MSc, BSc, Rebecca Griffiths, PhD, Nicola Cogan, Kongtana Trakarnsanga, MD PhD, David J. Anstee, PhD FRCPath, Ashley Mark Toye, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): SCI-5.
https://doi.org/10.1182/blood-2019-121117
Published: 2019
Journal Articles

Genetic Engineering in Hematopoietic Stem Cells for Gene Therapy of Hemoglobinopathies

Giuliana Ferrari, PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): SCI-23.
https://doi.org/10.1182/blood-2019-121119
Published: 2019
Journal Articles

Fully Closed, Large-Scale, and Clinical Grade Cell Sorting of Hematopoietic Stem Cell (HSC)-Enriched CD90 + Cells for Transplantation and Gene Therapy

Stefan Radtke, PhD, Margaret Cui, Anai M Perez, Yan-Yi Chan, Stefanie Schmuck, BS, Andrew J Berger, PhD, Thomas Eunson, BSc, Hans-Peter Kiem, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3246.
https://doi.org/10.1182/blood-2019-125969
Published: 2019