1 | Physiological role and mechanisms of action for a long noncoding haplotype region. (Xue H, Mishra MK, Liu Y, Liu P, Grzybowski M, Pandey R, Usa K, Vanden Avond MA, Bala N, Alli AA, Cowley AW Jr, Qiu Q, Greene AS, Rao S, O'Meara CC, Geurts AM, Liang M) Cell Rep 2025 Jun 24;44(6):115805 |
1 | The vectors went in two-by-two: Transduction efficiency and tolerability of dual and triple rAAV vector delivery following intravitreal injection for genome-editing applications. (Fehrman RL, Chern KJ, Stoltz KP, Lipinski DM) Exp Eye Res 2025 Feb;251:110223 1 Citation |
1 | RNA therapeutics to control fibrinolysis: review on applications in biology and medicine. (Ferraresso F, Leung J, Kastrup CJ) J Thromb Haemost 2024 Aug;22(8):2103-2114 2 Citations |
1 | Potent and uniform fetal hemoglobin induction via base editing. (Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS) Nat Genet 2023 Jul;55(7):1210-1220 38 Citations |
2 | Detection of chromosomal alteration after infusion of gene-edited allogeneic CAR T cells. (Sasu BJ, Opiteck GJ, Gopalakrishnan S, Kaimal V, Furmanak T, Huang D, Goswami A, He Y, Chen J, Nguyen A, Balakumaran A, Shah NN, Hamadani M, Bone KM, Prashad S, Bowen MA, Pertel T, Embree HD, Gidwani SG, Chang D, Moore A, Leonard M, Amado RG) Mol Ther 2023 Mar 01;31(3):676-685 19 Citations |
1 | Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations. (Woodard KJ, Doerfler PA, Mayberry KD, Sharma A, Levine R, Yen J, Valentine V, Palmer LE, Valentine M, Weiss MJ) Dis Model Mech 2022 Jun 01;15(6) 11 Citations |
1 | Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells. (Qin K, Huang P, Feng R, Keller CA, Peslak SA, Khandros E, Saari MS, Lan X, Mayuranathan T, Doerfler PA, Abdulmalik O, Giardine B, Chou ST, Shi J, Hardison RC, Weiss MJ, Blobel GA) Nat Genet 2022 Jun;54(6):874-884 26 Citations |
1 | The use of new CRISPR tools in cardiovascular research and medicine. (Nishiga M, Liu C, Qi LS, Wu JC) Nat Rev Cardiol 2022 Aug;19(8):505-521 43 Citations |
1 | A novel mouse model of type 2N VWD was developed by CRISPR/Cas9 gene editing and recapitulates human type 2N VWD. (Shi Q, Fahs SA, Mattson JG, Yu H, Perry CL, Morateck PA, Schroeder JA, Rapten J, Weiler H, Montgomery RR) Blood Adv 2022 May 10;6(9):2778-2790 5 Citations |
1 | Generation of 'designer erythroblasts' lacking one or more blood group systems from CRISPR/Cas9 gene-edited human-induced pluripotent stem cells. (Pandey P, Zhang N, Curtis BR, Newman PJ, Denomme GA) J Cell Mol Med 2021 Oct;25(19):9340-9349 10 Citations |
1 | Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin. (Doerfler PA, Feng R, Li Y, Palmer LE, Porter SN, Bell HW, Crossley M, Pruett-Miller SM, Cheng Y, Weiss MJ) Nat Genet 2021 Aug;53(8):1177-1186 34 Citations |
1 | Gene therapy for Fabry disease: Progress, challenges, and outlooks on gene-editing. (Domm JM, Wootton SK, Medin JA, West ML) Mol Genet Metab 2021;134(1-2):117-131 32 Citations |
1 | Whole-genome sequencing association analysis of quantitative red blood cell phenotypes: The NHLBI TOPMed program. (Hu Y, Stilp AM, McHugh CP, Rao S, Jain D, Zheng X, Lane J, Méric de Bellefon S, Raffield LM, Chen MH, Yanek LR, Wheeler M, Yao Y, Ren C, Broome J, Moon JY, de Vries PS, Hobbs BD, Sun Q, Surendran P, Brody JA, Blackwell TW, Choquet H, Ryan K, Duggirala R, Heard-Costa N, Wang Z, Chami N, Preuss MH, Min N, Ekunwe L, Lange LA, Cushman M, Faraday N, Curran JE, Almasy L, Kundu K, Smith AV, Gabriel S, Rotter JI, Fornage M, Lloyd-Jones DM, Vasan RS, Smith NL, North KE, Boerwinkle E, Becker LC, Lewis JP, Abecasis GR, Hou L, O'Connell JR, Morrison AC, Beaty TH, Kaplan R, Correa A, Blangero J, Jorgenson E, Psaty BM, Kooperberg C, Walton RT, Kleinstiver BP, Tang H, Loos RJF, Soranzo N, Butterworth AS, Nickerson D, Rich SS, Mitchell BD, Johnson AD, Auer PL, Li Y, Mathias RA, Lettre G, Pankratz N, Laurie CC, Laurie CA, Bauer DE, Conomos MP, Reiner AP, NHLBI Trans-Omics for Precision Medicine (TOPMed) Consortium) Am J Hum Genet 2021 May 06;108(5):874-893 25 Citations |
1 | Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing. (Leibowitz ML, Papathanasiou S, Doerfler PA, Blaine LJ, Sun L, Yao Y, Zhang CZ, Weiss MJ, Pellman D) Nat Genet 2021 Jun;53(6):895-905 366 Citations |
1 | The NIH Somatic Cell Genome Editing program. (Saha K, Sontheimer EJ, Brooks PJ, Dwinell MR, Gersbach CA, Liu DR, Murray SA, Tsai SQ, Wilson RC, Anderson DG, Asokan A, Banfield JF, Bankiewicz KS, Bao G, Bulte JWM, Bursac N, Campbell JM, Carlson DF, Chaikof EL, Chen ZY, Cheng RH, Clark KJ, Curiel DT, Dahlman JE, Deverman BE, Dickinson ME, Doudna JA, Ekker SC, Emborg ME, Feng G, Freedman BS, Gamm DM, Gao G, Ghiran IC, Glazer PM, Gong S, Heaney JD, Hennebold JD, Hinson JT, Khvorova A, Kiani S, Lagor WR, Lam KS, Leong KW, Levine JE, Lewis JA, Lutz CM, Ly DH, Maragh S, McCray PB Jr, McDevitt TC, Mirochnitchenko O, Morizane R, Murthy N, Prather RS, Ronald JA, Roy S, Roy S, Sabbisetti V, Saltzman WM, Santangelo PJ, Segal DJ, Shimoyama M, Skala MC, Tarantal AF, Tilton JC, Truskey GA, Vandsburger M, Watts JK, Wells KD, Wolfe SA, Xu Q, Xue W, Yi G, Zhou J, SCGE Consortium) Nature 2021 Apr;592(7853):195-204 92 Citations |
1 | Genome editing demonstrates that the -5 kb Nanog enhancer regulates Nanog expression by modulating RNAPII initiation and/or recruitment. (Agrawal P, Blinka S, Pulakanti K, Reimer MH Jr, Stelloh C, Meyer AE, Rao S) J Biol Chem 2021;296:100189 11 Citations |
1 | β-Arrestin1 and β-Arrestin2 Are Required to Support the Activity of the CXCL12/HMGB1 Heterocomplex on CXCR4. (D'Agostino G, Artinger M, Locati M, Perez L, Legler DF, Bianchi ME, Rüegg C, Thelen M, Marchese A, Rocchi MBL, Cecchinato V, Uguccioni M) Front Immunol 2020;11:550824 25 Citations |
1 | Stem Cell-Based Disease Modeling and Cell Therapy. (Bai X) Cells 2020 Sep 29;9(10) 24 Citations |
1 | Functions of Gtf2i and Gtf2ird1 in the developing brain: transcription, DNA binding and long-term behavioral consequences. (Kopp ND, Nygaard KR, Liu Y, McCullough KB, Maloney SE, Gabel HW, Dougherty JD) Hum Mol Genet 2020 Jun 03;29(9):1498-1519 15 Citations |
1 | Investigation of a dilated cardiomyopathy-associated variant in BAG3 using genome-edited iPSC-derived cardiomyocytes. (McDermott-Roe C, Lv W, Maximova T, Wada S, Bukowy J, Marquez M, Lai S, Shehu A, Benjamin I, Geurts A, Musunuru K) JCI Insight 2019 Nov 14;4(22) 36 Citations |
1 | Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. (Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ) Blood Adv 2019 Nov 12;3(21):3379-3392 133 Citations |
2 | Somatic Mutations of lats2 Cause Peripheral Nerve Sheath Tumors in Zebrafish. (Brandt ZJ, North PN, Link BA) Cells 2019 Aug 25;8(9) 11 Citations |
1 | Deletion of Tet proteins results in quantitative disparities during ESC differentiation partially attributable to alterations in gene expression. (Reimer M Jr, Pulakanti K, Shi L, Abel A, Liang M, Malarkannan S, Rao S) BMC Dev Biol 2019 Jul 08;19(1):16 9 Citations |
1 | Mapping Mammary Tumor Traits in the Rat. (Flister MJ, Joshi A, Bergom C, Rui H) Methods Mol Biol 2019;2018:249-267 4 Citations |
4 | Critical role of Jumonji domain of JMJD1C in MLL-rearranged leukemia. (Izaguirre-Carbonell J, Christiansen L, Burns R, Schmitz J, Li C, Mokry RL, Bluemn T, Zheng Y, Shen J, Carlson KS, Rao S, Wang D, Zhu N) Blood Adv 2019 May 14;3(9):1499-1511 23 Citations |
1 | Acyl-CoA Oxidases Fine-Tune the Production of Ascaroside Pheromones with Specific Side Chain Lengths. (Zhang X, Wang Y, Perez DH, Jones Lipinski RA, Butcher RA) ACS Chem Biol 2018 Apr 20;13(4):1048-1056 23 Citations |
4 | PITX2 deficiency and associated human disease: insights from the zebrafish model. (Hendee KE, Sorokina EA, Muheisen SS, Reis LM, Tyler RC, Markovic V, Cuturilo G, Link BA, Semina EV) Hum Mol Genet 2018 May 15;27(10):1675-1695 49 Citations |
1 | Functional characterization of zebrafish orthologs of the human Beta 3-Glucosyltransferase B3GLCT gene mutated in Peters Plus Syndrome. (Weh E, Takeuchi H, Muheisen S, Haltiwanger RS, Semina EV) PLoS One 2017;12(9):e0184903 14 Citations |
1 | Cos2/Kif7 and Osm-3/Kif17 regulate onset of outer segment development in zebrafish photoreceptors through distinct mechanisms. (Lewis TR, Kundinger SR, Pavlovich AL, Bostrom JR, Link BA, Besharse JC) Dev Biol 2017 May 15;425(2):176-190 21 Citations |
3 | Efficient Precision Genome Editing in iPSCs via Genetic Co-targeting with Selection. (Mitzelfelt KA, McDermott-Roe C, Grzybowski MN, Marquez M, Kuo CT, Riedel M, Lai S, Choi MJ, Kolander KD, Helbling D, Dimmock DP, Battle MA, Jou CJ, Tristani-Firouzi M, Verbsky JW, Benjamin IJ, Geurts AM) Stem Cell Reports 2017 Mar 14;8(3):491-499 26 Citations |
1 | EpCAM-Regulated Transcription Exerts Influences on Nanomechanical Properties of Endometrial Cancer Cells That Promote Epithelial-to-Mesenchymal Transition. (Hsu YT, Osmulski P, Wang Y, Huang YW, Liu L, Ruan J, Jin VX, Kirma NB, Gaczynska ME, Huang TH) Cancer Res 2016 Nov 01;76(21):6171-6182 47 Citations |
1 | Super-Enhancers at the Nanog Locus Differentially Regulate Neighboring Pluripotency-Associated Genes. (Blinka S, Reimer MH Jr, Pulakanti K, Rao S) Cell Rep 2016 Sep 27;17(1):19-28 83 Citations |
1 | Transcriptome Profiling of Patient-Specific Human iPSC-Cardiomyocytes Predicts Individual Drug Safety and Efficacy Responses In Vitro. (Matsa E, Burridge PW, Yu KH, Ahrens JH, Termglinchan V, Wu H, Liu C, Shukla P, Sayed N, Churko JM, Shao N, Woo NA, Chao AS, Gold JD, Karakikes I, Snyder MP, Wu JC) Cell Stem Cell 2016 Sep 01;19(3):311-25 131 Citations |
1 | Whole-Exome Sequencing Identifies Loci Associated with Blood Cell Traits and Reveals a Role for Alternative GFI1B Splice Variants in Human Hematopoiesis. (Polfus LM, Khajuria RK, Schick UM, Pankratz N, Pazoki R, Brody JA, Chen MH, Auer PL, Floyd JS, Huang J, Lange L, van Rooij FJ, Gibbs RA, Metcalf G, Muzny D, Veeraraghavan N, Walter K, Chen L, Yanek L, Becker LC, Peloso GM, Wakabayashi A, Kals M, Metspalu A, Esko T, Fox K, Wallace R, Franceschini N, Matijevic N, Rice KM, Bartz TM, Lyytikäinen LP, Kähönen M, Lehtimäki T, Raitakari OT, Li-Gao R, Mook-Kanamori DO, Lettre G, van Duijn CM, Franco OH, Rich SS, Rivadeneira F, Hofman A, Uitterlinden AG, Wilson JG, Psaty BM, Soranzo N, Dehghan A, Boerwinkle E, Zhang X, Johnson AD, O'Donnell CJ, Johnsen JM, Reiner AP, Ganesh SK, Sankaran VG) Am J Hum Genet 2016 Aug 04;99(2):481-8 36 Citations |
1 | Gene Editing: Practical Application in the Lab. (Geurts AM, Ekker SC) Hum Gene Ther 2016 Jun;27(6):417-8 1 Citation |