Human genetic diversity alters off-target outcomes of therapeutic gene editing von Cancellieri, Samuele, Zeng, Jing, Lin, Linda Yingqi, Tognon, Manuel, Nguyen, My Anh, Lin, Jiecong, Bombieri, Nicola, Maitland, Stacy A., Ciuculescu, Marioara-Felicia, Katta, Varun, Tsai, Shengdar Q., Armant, Myriam, Wolfe, Scot A., Giugno, Rosalba, Bauer, Daniel E., Pinello, Luca

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Deletion of Rhoh Accelerates Development of Diffuse Large B Cell Lymphoma in Bcl-6 Transgenic Mouse Model von Horiguchi, Hiroto, Ciuculescu, Marioara Felicia, Brendel, Christian, Williams, David A.

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Effects of BCL11A Shmir-Induced Post-Transcriptional Silencing on Distributions of HbF in Single-RBCs and Reticulocytes von Hebert, Nicolas, Esrick, Erica B., Armant, Myriam, Brendel, Christian, Ciuculescu, Marioara Felicia, Audureau, Etienne, Higgins, John M., Williams, David A., Bartolucci, Pablo

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Deletion of Murine Rhoh induces More Aggressive Diffuse Large B Cell Lymphoma (DLBCL) Via Interaction with Kaiso and Regulation of BCL-6 Expression von Horiguchi, Hiroto, Ciuculescu, Marioara Felicia, Troeger, Anja, Xu, Haiming, Brendel, Christian, Williams, David A.

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Human Genetic Diversity Alters Therapeutic Gene Editing Off-Target Outcomes von Lin, Linda Yingqi, Cancellieri, Samuele, Zeng, Jing, Masillo, Francesco, Nguyen, My Anh, Bombieri, Nicola, Maitland, Stacy A., Ciuculescu, Marioara-Felicia, Katta, Varun, Tsai, Shengdar Q., Armant, Myriam, Wolfe, Scot A., Giugno, Rosalba, Pinello, Luca, Bauer, Daniel E.

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Therapeutic Gene Editing of HSCs Ex Vivo without in Vitro Culture Avoids Genotoxicity, Simplifies Procedures, and Preserves Efficiency and Stemness von Zeng, Jing, Nguyen, My Anh, Ciuculescu, Marioara-Felicia, Mintzer, Esther, Liu, Pengpeng, Lin, Linda Yingqi, Rosanwo, Tolulope O, Verma, Archana, Neri, Nola, Maitland, Stacy A., Richter, Alden, Justus, David G., Clement, Kendell, Brendel, Christian, Pinello, Luca, Manis, John P, Armant, Myriam, Wolfe, Scot A., Bauer, Daniel E.

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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-Transcr... von Esrick, Erica B., Federico, Amy, Abriss, Daniela, Armant, Myriam, Boardman, Kari, Brendel, Christian, Ciuculescu, Marioara-Felicia, Daley, Heather, Dansereau, Colleen, Fernandes, Augustine, Heeney, Matthew M., Justus, David G., Kao, Pei-Chi, Kim, Annette S., Kohn, Donald B., Lehmann, Leslie E., Liu, Donghui, London, Wendy B., Manis, John P, Moore, Theodore B., Morris, Emily, Pellin, Danilo, Proeung, Ellen, Richard, Shanna, Roach, Gavin D., Shaw, Kit L., Terrazas, Dayna, White, Shanna L, Williams, David A.

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Combined +58 and +55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models von Zeng, Jing, Demirci, Selami, Nguyen, My Anh, Lin, Linda Yingqi, Maitland, Stacy A., Mintzer, Esther, Wu, Yuxuan, Pellin, Danilo, Tangprasittipap, Amornrat, Vong, Chokdee, Porter, Shaina N., Luk, Kevin, Liu, Pengpeng, Katta, Varun, Ciuculescu, Marioara-Felicia, Abriss, Daniela, Hsu, Jonathan, Uchida, Naoya, Essawi, Khaled, Donahue, Robert, Petri, Karl, Pattanayak, Vikram, Pinello, Luca, Brendel, Christian, Williams, David A, Manis, John P, Tsai, Shengdar Q., Pruett-Miller, Shondra M., Joung, J. Keith, Songdej, Duantida, Hongeng, Suradej, Armant, Myriam, Wolfe, Scot A., Tisdale, John F., Bauer, Daniel E.

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Validation of BCL11A As Therapeutic Target in Sickle Cell Disease: Results from the Adult Cohort of a Pilot/Feasibility Gene Therapy Trial Inducing Sustained Expression of Fetal He... von Esrick, Erica B., Achebe, Maureen, Armant, Myriam, Bartolucci, Pablo, Ciuculescu, Marioara Felicia, Daley, Heather, Dansereau, Colleen, Di Caprio, Giuseppe, Goncalves, Bronner, Hebert, Nicolas, Heeney, Matthew M, Higgins, John M., Lehmann, Leslie E., Manis, John P, Negre, Olivier, Nikiforow, Sarah, Schonbrun, Ethan, Wood, David K., Williams, David A.

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Flipping the Switch: Initial Results of Genetic Targeting of the Fetal to Adult Globin Switch in Sickle Cell Patients von Esrick, Erica B., Brendel, Christian, Manis, John P, Armant, Myriam A, Negre, Helene, Dansereau, Colleen, Ciuculescu, Marioara Felicia, Patriarca, Stephanie, Mackinnon, Brenda, Daley, Heather, Richard, Shanna, Abriss, Daniela, Maxwell, Renee, Braunewell, Stephen, Christiansen, Lauryn, Nikiforow, Sarah, Ritz, Jerome, Achebe, Maureen, Negre, Olivier, Lehmann, Leslie E., Heeney, Matthew M, Biffi, Alessandra, Williams, David A.

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