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Luca Pinello
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Journal Articles

Therapeutic Gene Editing of HSCs Ex Vivo without in Vitro Culture Avoids Genotoxicity, Simplifies Procedures, and Preserves Efficiency and Stemness

Jing Zeng, My Anh Nguyen, Marioara-Felicia Ciuculescu, Esther Mintzer, Pengpeng Liu, Linda Yingqi Lin, Tolulope O Rosanwo, Archana Verma, Nola Neri, Stacy A. Maitland, Alden Richter, David G. Justus, Kendell Clement, Christian Brendel, Luca Pinello, John P Manis, Myriam Armant, Scot A. Wolfe, Daniel E. Bauer
Journal: Blood
Blood (2022) 140 (Supplement 1): 10671–10672.
https://doi.org/10.1182/blood-2022-169900
Published: 2022
Journal Articles

Identification of a Novel Epigenetic Mechanism of MYC Deregulation in Smoldering and Newly Diagnosed Multiple Myeloma Patients

Mahshid Rahmat, Kendell Clement, Romanos Sklavenitis-Pistofidis, Rohan Kodgule, Charles Fulco, Jean-Baptiste Alberge, Cody J. Boehner, Michael P. Agius, Elizabeth Morgan Kitzenberg, David Dorfman, Russell J. H. Ryan, Luca Pinello, Irene M. Ghobrial
Journal: Blood
Blood (2021) 138 (Supplement 1): 504.
https://doi.org/10.1182/blood-2021-150734
Published: 2021
Journal Articles

Human Genetic Diversity Alters Therapeutic Gene Editing Off-Target Outcomes

Linda Yingqi Lin, Samuele Cancellieri, Jing Zeng, Francesco Masillo, My Anh Nguyen, Nicola Bombieri, Stacy A. Maitland, Marioara-Felicia Ciuculescu, Varun Katta, Shengdar Q. Tsai, Myriam Armant, Scot A. Wolfe, Rosalba Giugno, Luca Pinello, Daniel E. Bauer
Journal: Blood
Blood (2021) 138 (Supplement 1): 3993.
https://doi.org/10.1182/blood-2021-152429
Published: 2021
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

ZNF410 Represses Fetal Globin By Devoted Control of CHD4/NuRD

Divya S Vinjamur, PhD, Qiuming Yao, PhD, Mitchel A. Cole, BS, Connor McGuckin, Chunyan Ren, PhD, Jing Zeng, MD, Mir Hossain, PhD, Luca Pinello, PhD, Daniel E Bauer, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 1.
https://doi.org/10.1182/blood-2020-142134
Published: 2020
Journal Articles

Multiplexed CRISPR In Vivo Editing of CLL Loss-of-Function Lesions Models Transformation of Chronic Lymphocytic Leukemia into Richter's Syndrome

Elisa Ten Hacken, PhD, Tomasz Sewastianik, PhD, Robert A. Redd, MS, Geoffrey Fell, MS, Mohamed Uduman, PhD, Michaela Gruber, MD PhD, Shanye Yin, PhD, Kendell Clement, PhD, Erin Michelle Parry, MD PhD, Shuqiang Li, PhD, Maria Hernandez-Sanchez, PhD, Leah Billington, BA, Elizabeth Witten, Kaitlyn J Baranowski, Lili Wang, MDPhD, Luca Pinello, PhD, Kenneth J. Livak, PhD, Donna S. Neuberg, ScD, Ruben D. Carrasco, MD PhD, Catherine J. Wu, MD
Journal: Blood
Blood (2020) 136 (Supplement 1): 2–3.
https://doi.org/10.1182/blood-2020-137130
Published: 2020
Journal Articles

Interrogation of Individual CLL Loss-of-Function Lesions By CRISPR In Vivo Editing Reveals Common and Unique Pathway Alterations

Elisa Ten Hacken, PhD, Shanye Yin, PhD, Kendell Clement, PhD, Robert A. Redd, MS, Maria Hernandez-Sanchez, PhD, Shuqiang Li, PhD, Michaela Gruber, MD PhD, Heather Joyal, Geoffrey Fell, MS, Shu Wang, Jose Jacob, James Flynn, David Ruff, Keith W. Jones, Lili Wang, MD PhD, Donna S Neuberg, ScD, Luca Pinello, PhD, Kenneth J. Livak, PhD, Ruben D. Carrasco, MD PhD, Catherine J. Wu, MD
Journal: Blood
Blood (2019) 134 (Supplement_1): 684.
https://doi.org/10.1182/blood-2019-127673
Published: 2019
Journal Articles

CRISPR-Cas9 Screen Identifies XPO7 As a Potential Therapeutic Target for TP53 -Mutated AML

Yuichiro Semba, MD PhD, Takuji Yamauchi, MD PhD, Fumihiko Nakao, MD, Jumpei Nogami, PhD, Matthew C. Canver, MD PhD, Luca Pinello, PhD, Daniel E. Bauer, MD PhD, Koichi Akashi, MD PhD, Takahiro Maeda, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3784.
https://doi.org/10.1182/blood-2019-129623
Published: 2019
Journal Articles

Gene Editing ELANE in Human Hematopoietic Stem and Progenitor Cells Reveals Disease Mechanisms and Therapeutic Strategies for Severe Congenital Neutropenia

Shuquan Rao, PhD, Josias Brito-Frazao, Anna V. Serbin, Qiuming Yao, PhD, Kevin Luk, Yuxuan Wu, PhD, Jing Zeng, MD, Chunyan Ren, PhD, Ruth Watkinson, Myriam Armant, PhD, Luca Pinello, PhD, Akiko Shimamura, MD PhD, Benhur Lee, Scot A. Wolfe, PhD, Peter E. Newburger, MD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 3.
https://doi.org/10.1182/blood-2019-131073
Published: 2019
Journal Articles

Paics, a De Novo Purine Synthetic Enzyme, Is a Novel Target for AML Therapy

Takuji Yamauchi, MD PhD, Kohta Miyawaki, MD, Yuichiro Semba, MD PhD, Fumihiko Nakao, MD, Takeshi Sugio, MD PhD, Kensuke Sasaki, MD, Jumpei Nogami, PhD, Matthew C. Canver, MD PhD, Simon Osborne, PhD, Luca Pinello, PhD, Deborah Taylor, PhD, Daniel E. Bauer, MD PhD, Koichi Akashi, MD PhD, Takahiro Maeda, MD PhD
Journal: Blood
Blood (2019) 134 (Supplement_1): 1390.
https://doi.org/10.1182/blood-2019-127047
Published: 2019
Journal Articles

Editing aberrant splice sites efficiently restores β-globin expression in β-thalassemia

Brief Report
Shuqian Xu, Kevin Luk, Qiuming Yao, Anne H. Shen, Jing Zeng, Yuxuan Wu, Hong-Yuan Luo, Christian Brendel, Luca Pinello, David H. K. Chui, Scot A. Wolfe, Daniel E. Bauer
Journal: Blood
Blood (2019) 133 (21): 2255–2262.
https://doi.org/10.1182/blood-2019-01-895094
Published: 2019
Includes: Supplemental data
Journal Articles

Comprehensive Integrated Genomic Perturbations Reveal Molecular Mechanisms of Red Blood Cell Trait Associations

Yuxuan Wu, PhD, Mitchel A. Cole, BS, Abdou Mousas, Jing Zeng, MD, Qiuming Yao, PhD, Divya Vinjamur, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Luca Pinello, PhD, Guillame Lettre, PhD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 532.
https://doi.org/10.1182/blood-2018-99-120229
Published: 2018
Journal Articles

Highly Efficient Therapeutic Gene Editing of BCL11A enhancer in Human Hematopoietic Stem Cells from ß-Hemoglobinopathy Patients for Fetal Hemoglobin Induction

Jing Zeng, MD, Daniel E. Bauer, MD PhD, Yuxuan Wu, PhD, Benjamin P Roscoe, Pengpeng Liu, Qiuming Yao, PhD, Cicera Lazzarrotto, M. Kendell Clement, Mitchel A. Cole, BS, Kevin Luk, Cristina Baricordi, Erica B. Esrick, MD, John P Manis, MD, David Dorfma, David A. Williams, MD, Alessandra Biffi, MD PhD, Carlo Brugnara, MD, Luca Biasco, Christian Brendel, PhD, Luca Pinello, PhD, Shengdar Tsai, PhD, Scot A. Wolfe
Journal: Blood
Blood (2018) 132 (Supplement 1): 3482.
https://doi.org/10.1182/blood-2018-99-119365
Published: 2018
Journal Articles

Rational Targeting of a NuRD Sub-Complex for Fetal Hemoglobin Induction Following Comprehensive in Situ Mutagenesis

Mir Hossain, PhD, Falak Sher, PhD, Davide Seruggia, PhD, Vivien Schoonenberg, BS, Mitchel A. Cole, BS, Paolo Cifani, PhD, Laura Dassama, PhD, Claudio Macias-Trevino, BS, Matthew C. Canver, MD PhD, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Luca Pinello, PhD, Takahiro Maeda, MD PhD, Alex Kentsis, MD PhD, Stuart H. Orkin, MD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2342.
https://doi.org/10.1182/blood-2018-99-117043
Published: 2018
Journal Articles

Genome-Wide CRISPR/Cas9 Screen Reveals That the Dcps Scavenger Decapping Enzyme Is Essential for AML Cell Survival

Takuji Yamauchi, MDPhD, Takeshi Masuda, PhD, Matthew C. Canver, BS, Michael Seiler, PhD, Mohammad Shboul, Manami Maeda, MDPhD, Vivien Schoonenberg, BS, Mitchel A. Cole, BS, Claudio Macias-Trevino, BS, Yuichiro Semba, MD, Yuichi Ishikawa, MD PhD, Michitaka Nakano, MD, Fumio Arai, PhD, Stuart Orkin, Bruno Reversade, Silvia Buonamici, PhD, Luca Pinello, PhD, Koichi Akashi, MD PhD, Daniel E. Bauer, MD PhD, Takahiro Maeda, MDPhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 782.
https://doi.org/10.1182/blood.V130.Suppl_1.782.782
Published: 2017
Journal Articles

High-Resolution CRISPR Functional Genomics Reveals Critical Vulnerabilities of the NuRD Complex for Fetal Hemoglobin Control

Falak Sher, PhD, Vivien Schoonenberg, BS, Mitchel A. Cole, BS, Claudio Macias-Trevino, BS, Paolo Cifani, PhD, Patrick G Schupp, BS, Matthew C. Canver, BS, Ryo Kurita, PhD, Yukio Nakamura, MD PhD, Stuart Orkin, Alex Kentsis, MD PhD, Luca Pinello, PhD, Takahiro Maeda, MDPhD, Daniel E. Bauer, MD PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 286.
https://doi.org/10.1182/blood.V130.Suppl_1.286.286
Published: 2017
Journal Articles

Crispr-Cas9 Saturating Mutagenesis Reveals an Achilles Heel in the BCL11A Erythroid Enhancer for Fetal Hemoglobin Induction (by Genome Editing)

Daniel E. Bauer, MD, PhD, Matthew C. Canver, B.S., Elenoe C. Smith, PhD, Falak Sher, PhD, Luca Pinello, PhD, Neville E. Sanjana, PhD, Ophir Shalem, PhD, Diane D. Chen, B.S., Patrick G. Schupp, B.S., Divya S. Vinjamur, PhD, Sara P. Garcia, PhD, Sidinh Luc, PhD, Ryo Kurita, PhD, Yukio Nakamura, MD, PhD, Yuko Fujiwara, PhD, Takahiro Maeda, MD, PhD, Guo-Cheng Yuan, PhD, Guillaume Lettre, PhD, Feng Zhang, PhD, Stuart H. Orkin, MD
Journal: Blood
Blood (2015) 126 (23): 638.
https://doi.org/10.1182/blood.V126.23.638.638
Published: 2015
Journal Articles

Developmental Control of Polycomb Subunit Composition Mediates a Switch to Non-Canonical Functions during Hematopoiesis

Jian Xu, PhD, Zhen Shao, PhD, Dan Li, Huafeng Xie, PhD, Woojin Kim, PhD, Jialiang Huang, PhD, Luca Pinello, PhD, Kimberly Glass, PhD, Guo-Cheng Yuan, PhD, Stuart H. Orkin, MD
Journal: Blood
Blood (2014) 124 (21): 241.
https://doi.org/10.1182/blood.V124.21.241.241
Published: 2014
Journal Articles

Fine-Mapping and Genome Editing Reveal An Essential Erythroid Enhancer At The HbF-Associated BCL11A Locus

Daniel E. Bauer, MD, PHD, Sophia C. Kamran, MD, Samuel Lessard, BS, Jian Xu, PhD, Yuko Fujiwara, PhD, Carrie Lin, BS, Zhen Shao, PhD, Matthew C. Canver, BS, Elenoe C. Smith, PhD, Luca Pinello, PhD, Peter J. Sabo, PhD, Jeff Vierstra, BS, Richard A. Voit, BS, Guo-Cheng Yuan, PhD, Matthew H. Porteus, MD, PhD, John Stamatoyannopoulos, MD, Guillaume Lettre, PhD, Stuart H. Orkin, MD
Journal: Blood
Blood (2013) 122 (21): 437.
https://doi.org/10.1182/blood.V122.21.437.437
Published: 2013
Journal Articles

Genomic Determinants of Human Fetal and Adult Erythroid Gene Expression

Jian Xu, PhD, Zhen Shao, PhD, Kimberly Glass, PhD, Daniel E. Bauer, MD, PhD, Luca Pinello, PhD, Ben Van Handel, PhD, Serena Hou, John A. Stamatoyannopoulos, MD, Hanna K. A. Mikkola, MD, PhD, Guo-Cheng Yuan, PhD, Stuart H. Orkin, MD
Journal: Blood
Blood (2012) 120 (21): 1227.
https://doi.org/10.1182/blood.V120.21.1227.1227
Published: 2012