Selected Publications
Feng R, Mayuranathan T, Huang P, Doerfler PA, Li Y, Yao Y, Zhang J, Palmer LE, Mayberry K, Christakopoulos GE, Xu P,Li C, Cheng Y, Blobel GA, Simon MC, Weiss MJ. Activation of γ-globin expression by hypoxia-inducible factor 1α. Nature Oct;610(7933):783-790, 2022. doi: 10.1038/s41586-022-05312-w. Epub 2022 Oct 12. PMID: 36224385
Woodard KJ, Doerfler PA, Mayberry KD, Sharma A, Levine R, Yen J, Valentine V, Palmer LE, Valentine M, Weiss MJ. Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations. Dis Model Mech Jun 1;15(6):dmm049463, 2022. PMID: 35793591; PMCID: PMC9277148.
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; PMCID: PMC9203980
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 2;53(8):1177-1186, 2021. PMID: 34341563; PMCID: PMC8610173
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; PMCID: PMC8192433. †‡Denotes equal contribution
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; PMCID: PMC8262557. *Denotes equal contribution
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; PMCID: PMC6855127. *Denotes equal contribution
Last update: December 2022