Biblio

Author Title [ Type(Desc)] Year
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Journal Article
Pinello L, Canver MC, Hoban MD, Orkin SH, Kohn DB, Bauer DE, Yuan G-C. Analyzing CRISPR genome-editing experiments with CRISPResso. Nat Biotechnol. 2016;34(7):695-697.
Gehrke JM, Cervantes O, M Clement K, Wu Y, Zeng J, Bauer DE, Pinello L, J Joung K. An APOBEC3A-Cas9 base editor with minimized bystander and off-target activities. Nat Biotechnol. 2018.
Tao J, Bauer DE, Chiarle R. Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing. Nat Commun. 2023;14(1):212.
Zeng J, Casirati G, Nguyen MAnh, Genovese P, Bauer DE. Base Editing of Human Hematopoietic Stem Cells. Methods Mol Biol. 2023;2606:43-62.
Demirci S, Zeng J, Wu Y, Uchida N, Shen AH, Pellin D, Gamer J, Yapundich M, Drysdale C, Bonanno J, et al. BCL11A enhancer edited hematopoietic stem cells persist in rhesus monkeys without toxicity. J Clin Invest. 2020.
Liu B, Brendel C, Vinjamur DS, Zhou Y, Harris C, McGuinness M, Manis JP, Bauer DE, Xu H, Williams DA. Development of a double shmiR lentivirus effectively targeting both BCL11A and ZNF410 for enhanced induction of fetal hemoglobin to treat β-hemoglobinopathies. Mol Ther. 2022.
Rao S, Yao Y, de Brito JSoares, Yao Q, Shen AH, Watkinson RE, Kennedy AL, Coyne S, Ren C, Zeng J, et al. Dissecting ELANE neutropenia pathogenicity by human HSC gene editing. Cell Stem Cell. 2021.
Kim SS, Kycia I, Karski M, Ma RK, Bordt EA, Kwan J, Karki A, Winter E, Aktas RG, Wu Y, et al. DNAJB1-PRKACA in HEK293T cells induces LINC00473 overexpression that depends on PKA signaling. PLoS One. 2022;17(2):e0263829.
Casirati G, Cosentino A, Mucci A, Mahmoud MSalah, Zabala IUgarte, Zeng J, Ficarro SB, Klatt D, Brendel C, Rambaldi A, et al. Epitope editing enables targeted immunotherapy of acute myeloid leukaemia. Nature. 2023.
Demirci S, Khan MBN, Hinojosa G, Le A, Leonard A, Essawi K, Gudmundsdottir B, Liu X, Zeng J, Inam Z, et al. Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models. Cytotherapy. 2024.
Levesque S, Bauer DE. Gene correction for sickle cell disease hits its prime. Nat Biomed Eng. 2023;7(5):605-606.
Métais J-Y, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, et al. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv. 2019;3(21):3379-3392.
Bauer DE, Orkin SH. Hemoglobin switching's surprise: the versatile transcription factor BCL11A is a master repressor of fetal hemoglobin. Curr Opin Genet Dev. 2015;33:62-70.
Cancellieri S, Zeng J, Lin LYingqi, Tognon M, Nguyen MAnh, Lin J, Bombieri N, Maitland SA, Ciuculescu M-F, Katta V, et al. Human genetic diversity alters off-target outcomes of therapeutic gene editing. Nat Genet. 2022.
Yan J, Oyler-Castrillo P, Ravisankar P, Ward CC, Levesque S, Jing Y, Simpson D, Zhao A, Li H, Yan W, et al. Improving prime editing with an endogenous small RNA-binding protein. Nature. 2024.
Brendel C, Guda S, Renella R, Bauer DE, Canver MC, Kim Y-J, Heeney MM, Klatt D, Fogel J, Milsom MD, et al. Lineage-specific BCL11A knockdown circumvents toxicities and reverses sickle phenotype. J Clin Invest. 2016.
Tesio N, Bauer DE. Molecular Basis and Genetic Modifiers of Thalassemia. Hematol Oncol Clin North Am. 2023;37(2):273-299.
Bauer DE. Pervasive donor DNA integration defies precision gene editing of hematopoietic stem cells. Cell Stem Cell. 2022;29(10):1426-1427.
Sher F, Hossain M, Seruggia D, Schoonenberg VAC, Yao Q, Cifani P, Dassama LMK, Cole MA, Ren C, Vinjamur DS, et al. Rational targeting of a NuRD subcomplex guided by comprehensive in situ mutagenesis. Nat Genet. 2019.
Marion W, Koppe T, Chen C-C, Wang D, Frenis K, Fierstein S, Sensharma P, Aumais O, Peters M, Ruiz-Torres S, et al. RUNX1 mutations mitigate quiescence to promote transformation of hematopoietic progenitors in Fanconi anemia. Leukemia. 2023.
Nagpal N, Wang J, Zeng J, Lo E, Moon DH, Luk K, Braun RO, Burroughs LM, Keel SB, Reilly C, et al. Small-Molecule PAPD5 Inhibitors Restore Telomerase Activity in Patient Stem Cells. Cell Stem Cell. 2020.
Hinze L, Pfirrmann M, Karim S, Degar J, McGuckin C, Vinjamur D, Sacher J, Stevenson KE, Neuberg DS, Orellana E, et al. Synthetic Lethality of Wnt Pathway Activation and Asparaginase in Drug-Resistant Acute Leukemias. Cancer Cell. 2019;35(4):664-676.e7.
Liao J, Chen S, Hsiao S, Jiang Y, Yang Y, Zhang Y, Wang X, Lai Y, Bauer DE, Wu Y. Therapeutic adenine base editing of human hematopoietic stem cells. Nat Commun. 2023;14(1):207.
Masuda T, Wang X, Maeda M, Canver MC, Sher F, Funnell APW, Fisher C, Suciu M, Martyn GE, Norton LJ, et al. Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin. Science. 2016;351(6270):285-289.