Selected Publications
Crossley M, Christakopoulos GE, Weiss MJ. Effective therapies for sickle cell disease: are we there yet? Trends Genet Aug 4, 2022. [Epub ahead of print] Review. PMID: 35934593
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. Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells. Nat Genet Jun;54(6):874-884, 2022. PMID: 35618846
Topfer SK, Feng R, Huang P, Ly LC, Martyn GE, Blobel GA, Weiss MJ, Quinlan KGR, Crossley M. Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression. Blood Apr 7;139(14):2107-2118, 2022. PMID: 35090172
Keller TCS 4th, Lechauve C, Keller AS, Brooks S, Weiss MJ, Columbus L, Ackerman H, Cortese-Krott MM, Isakson BE. The role of globins in cardiovascular physiology. Physiol Rev Apr 1;102(2):859-892, 2022. Review. PMID: 34486392
Kiger L, Keith J, Freiwan A, Fernandez AG, Tillman H, Isakson BE, Weiss MJ, Lechauve C. Redox-Regulation of α-Globin in Vascular Physiology. Antioxidants (Basel) Jan 14;11(1), 2022. PMID: 35052663
Ji X, Jha A, Humenik J, Ghanem LR, Kromer A, Duncan-Lewis C, Traxler E, Weiss MJ, Barash Y, Liebhaber SA. RNA-Binding Proteins PCBP1 and PCBP2 Are Critical Determinants of Murine Erythropoiesis. Mol Cell Biol Aug 24;41(9):e0066820, 2021. PMID: 34180713
Doerfler PA, Feng R, Li Y, Palmer LE, Porter SN, Bell HW, Crossley M, Pruett-Miller SM, Cheng Y, Weiss MJ. Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin. Nat Genet Aug;53(8):1177-1186, 2021. PMID: 34341563
Rampersaud E, Kang G, Palmer LE, Rashkin SR, Wang S, Bi W, Alberts NM, Anghelescu D, Barton M, Birch K, Boulos N, Brandow AM, Brooke RJ, Chang TC, Chen W, Cheng Y, Ding J, Easton J, Hodges JR, Kanne CK, Levy S, Mulder H, Patel AP, Puri L, Rosencrance C, Rusch M, Sapkota Y, Sioson E, Sharma A, Tang X, Thrasher A, Wang W, Yao Y, Yasui Y, Yergeau D, Hankins JS, Sheehan VA, Downing JR, Estepp JH, Zhang J, DeBaun M, Wu G, Weiss MJ. A polygenic score for acute vaso-occlusive pain in pediatric sickle cell disease. Blood Adv Jul 27;5(14):2839-2851, 2021. PMID: 34283174
Rashkin SR, Rampersaud E, Kang G, Ataga KI, Hankins JS, Wang W, Estepp JH, Weiss MJ, Lebensburger J, Zahr RS. Generalization of a genetic risk score for time to first albuminuria in children with sickle cell anaemia: SCCRIP cohort study results. Br J Haematol Jul;194(2):469-473, 2021. PMID: 34137022
Newby GA, Yen JS, Woodard KJ, Mayuranathan T, Lazzarotto CR, Li Y, Sheppard-Tillman H, Porter SN, Yao Y, Mayberry K, Everette KA, Jang Y, Podracky CJ, Thaman E, Lechauve C, Sharma A, Henderson JM, Richter MF, Zhao KT, Miller SM, Wang T, Koblan LW, McCaffrey AP, Tisdale JF, Kalfa TA, Pruett-Miller SM, Tsai SQ, Weiss MJ, Liu DR. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature Jul;595(7866):295-302, 2021. PMID: 34079130
Leibowitz ML, Papathanasiou S, Doerfler PA, Blaine LJ, Sun L, Yao Y, Zhang CZ, Weiss MJ, Pellman D. Chromothripsis as an on-target consequence of CRISPR-Cas9 genome editing. Nat Genet Jun;53(6):895-905, 2021. PMID: 33846636
Cheng L, Li Y, Qi Q, Xu P, Feng R, Palmer L, Chen J, Wu R, Yee T, Zhang J, Yao Y, Sharma A, Hardison RC, Weiss MJ, Cheng Y. Single-nucleotide-level mapping of DNA regulatory elements that control fetal hemoglobin expression. Nat Genet Jun;53(6):869-880, 2021. PMID: 33958780
Leonard A, Sharma A, Uchida N, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease. Blood Adv May 11;5(9):2403-2411, 2021. PMID: 33956057
McLeod C, Gout AM, Zhou X, Thrasher A, Rahbarinia D, Brady SW, Macias M, Birch K, Finkelstein D, Sunny J, Mudunuri R, Orr BA, Treadway M, Davidson B, Ard TK, Chiao A, Swistak A, Wiggins S, Foy S, Wang J, Sioson E, Wang S, Michael JR, Liu Y, Ma X, Patel A, Edmonson MN, Wilkinson MR, Frantz AM, Chang TC, Tian L, Lei S, Islam SMA, Meyer C, Thangaraj N, Tater P, Kandali V, Ma S, Nguyen T, Serang O, McGuire I, Robison N, Gentry D, Tang X, Palmer LE, Wu G, Suh E, Tanner L, McMurry J, Lear M, Pappo AS, Wang Z, Wilson CL, Cheng Y, Meshinchi S, Alexandrov LB, Weiss MJ, Armstrong GT, Robison LL, Yasui Y, Nichols KE, Ellison DW, Bangur C, Mullighan CG, Baker SJ, Dyer MA, Miller G, Newman S, Rusch M, Daly R, Perry K, Downing JR, Zhang J. St. Jude Cloud: A Pediatric Cancer Genomic Data-Sharing Ecosystem. Cancer Discov May;11(5):1082-1099, 2021. PMID: 33408242
Doerfler PA, Sharma A, Porter JS, Zheng Y, Tisdale JF, Weiss MJ. Genetic therapies for the first molecular disease. J Clin Invest Apr 15;131(8), 2021. PMID: 33855970
Qi Q, Cheng L, Tang X, He Y, Li Y, Yee T, Shrestha D, Feng R, Xu P, Zhou X, Pruett-Miller S, Hardison RC, Weiss MJ, Cheng Y. Dynamic CTCF binding directly mediates interactions among cis-regulatory elements essential for hematopoiesis. Blood Mar 11;137(10):1327-1339, 2021. PMID: 33512425
Lan X, Ren R, Feng R, Ly LC, Lan Y, Zhang Z, Aboreden N, Qin K, Horton JR, Grevet JD, Mayuranathan T, Abdulmalik O, Keller CA, Giardine B, Hardison RC, Crossley M, Weiss MJ, Cheng X, Shi J, Blobel GA. ZNF410 Uniquely Activates the NuRD Component CHD4 to Silence Fetal Hemoglobin Expression. Mol Cell Jan 21;81(2):239-254.e8, 2021. PMID: 33301730
Xu P, Scott DC, Xu B, Yao Y, Feng R, Cheng L, Mayberry K, Wang YD, Bi W, Palmer LE, King MT, Wang H, Li Y, Fan Y, Alpi AF, Li C, Peng J, Papizan J, Pruett-Miller SM, Spallek R, Bassermann F, Cheng Y, Schulman BA, Weiss MJ. FBXO11-mediated proteolysis of BAHD1 relieves PRC2-dependent transcriptional repression in erythropoiesis. Blood Jan 14;137(2):155-167, 2021. PMID: 33156908
Karayel Ö, Xu P, Bludau I, Velan Bhoopalan S, Yao Y, Ana Rita FC, Santos A, Schulman BA, Alpi AF, Weiss MJ, Mann M. Integrative proteomics reveals principles of dynamic phosphosignaling networks in human erythropoiesis. Mol Syst Biol Dec;16(12):e9813, 2020. PMID: 33259127
Uchida N, Leonard A, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Sharma A, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor. Haematologica Oct 1;105(10):e497, 2020. PMID: 33054091
Chang TC, Haupfear KM, Yu J, Rampersaud E, Sheehan VA, Flanagan JM, Hankins JS, Weiss MJ, Wu G, Vege S, Westhoff CM, Chou ST, Zheng Y. A novel algorithm comprehensively characterizes human RH genes using whole-genome sequencing data. Blood Adv Sep 22;4(18):4347-4357, 2020. PMID: 32915977
Bhoopalan SV, Huang LJ, Weiss MJ. Erythropoietin regulation of red blood cell production: from bench to bedside and back. F1000Res 9, 2020. Review. PMID: 32983414
Xiang G, Keller CA, Heuston E, Giardine BM, An L, Wixom AQ, Miller A, Cockburn A, Sauria MEG, Weaver K, Lichtenberg J, Göttgens B, Li Q, Bodine D, Mahony S, Taylor J, Blobel GA, Weiss MJ, Cheng Y, Yue F, Hughes J, Higgs DR, Zhang Y, Hardison RC. An integrative view of the regulatory and transcriptional landscapes in mouse hematopoiesis. Genome Res Mar;30(3):472-484, 2020. PMID: 32132109
Hardison RC, Zhang Y, Keller CA, Xiang G, Heuston EF, An L, Lichtenberg J, Giardine BM, Bodine D, Mahony S, Li Q, Yue F, Weiss MJ, Blobel GA, Taylor J, Hughes J, Higgs DR, Göttgens B. Systematic integration of GATA transcription factors and epigenomes via IDEAS paints the regulatory landscape of hematopoietic cells. IUBMB Life Jan;72(1):27-38, 2020. Review. PMID: 31769130
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. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv Nov 12;3(21):3379-3392, 2019. PMID: 31698466
Zahr RS, Rampersaud E, Kang G, Weiss MJ, Wu G, Davis RL, Hankins JS, Estepp JH, Lebensburger J. Children with sickle cell anemia and APOL1 genetic variants develop albuminuria early in life. Haematologica Sep;104(9):e385-e387, 2019. PMID: 30890594
Lechauve C, Keith J, Khandros E, Fowler S, Mayberry K, Freiwan A, Thom CS, Delbini P, Romero EB, Zhang J, Motta I, Tillman H, Cappellini MD, Kundu M, Weiss MJ. The autophagy-activating kinase ULK1 mediates clearance of free α-globin in β-thalassemia. Sci Transl Med Aug 21;11(506), 2019. PMID: 31434755
Labuhn M, Perkins K, Matzk S, Varghese L, Garnett C, Papaemmanuil E, Metzner M, Kennedy A, Amstislavskiy V, Risch T, Bhayadia R, Samulowski D, Hernandez DC, Stoilova B, Iotchkova V, Oppermann U, Scheer C, Yoshida K, Schwarzer A, Taub JW, Crispino JD, Weiss MJ, Hayashi Y, Taga T, Ito E, Ogawa S, Reinhardt D, Yaspo ML, Campbell PJ, Roberts I, Constantinescu SN, Vyas P, Heckl D, Klusmann JH. Mechanisms of Progression of Myeloid Preleukemia to Transformed Myeloid Leukemia in Children with Down Syndrome. Cancer Cell Aug 12;36(2):123-138.e10, 2019. PMID: 31303423
Xu P, Palmer LE, Lechauve C, Zhao G, Yao Y, Luan J, Vourekas A, Tan H, Peng J, Schuetz JD, Mourelatos Z, Wu G, Weiss MJ, Paralkar VR. Regulation of gene expression by miR-144/451 during mouse erythropoiesis. Blood Jun 6;133(23):2518-2528, 2019. PMID: 30971389
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1. N Engl J Med Apr 18;380(16):1525-1534, 2019. PMID: 30995372
Tasian SK, Casas JA, Posocco D, Gandre-Babbe S, Gagne AL, Liang G, Loh ML, Weiss MJ, French DL, Chou ST. Mutation-specific signaling profiles and kinase inhibitor sensitivities of juvenile myelomonocytic leukemia revealed by induced pluripotent stem cells. Leukemia Jan;33(1):181-190, 2019. PubMed PMID: 29884903
Last update: August 2022