Medical College of Wisconsin
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Phillip A. Doerfler PhD

Phillip A. Doerfler PhD profile photo picture

Assistant Professor

Institution: Medical College of Wisconsin
Department: Cell Biology, Neurobiology and Anatomy
Program: Administration

View Curriculum Vitae

Publications (18)

  • Hydroxyurea reduces the levels of the fetal globin gene repressors ZBTB7A/LRF and BCL11A in erythroid cells in vitro. (Martyn GE, Doerfler PA, Yao Y, Quinlan KGR, Weiss MJ, Crossley M) J Sick Cell Dis 2024;1(1):yoae008 PMID: 40304012 PMCID: PMC12039817 04/30/2025    
  • 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 PMID: 37400614 PMCID: PMC10722557 SCOPUS ID: 2-s2.0-85163718652 07/04/2023       38 Citations
  • Activation of γ-globin expression by hypoxia-inducible factor 1α. (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) Nature 2022 Oct;610(7933):783-790 PMID: 36224385 PMCID: PMC9773321 SCOPUS ID: 2-s2.0-85139619738 10/13/2022       38 Citations
  • 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) PMID: 35793591 PMCID: PMC9277148 SCOPUS ID: 2-s2.0-85133634578 07/07/2022       11 Citations
  • Publisher Correction: 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):906 PMID: 35650318 SCOPUS ID: 2-s2.0-85131336665 06/02/2022    
  • 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 PMID: 35618846 PMCID: PMC9203980 SCOPUS ID: 2-s2.0-85130717494 05/27/2022       26 Citations
  • 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 PMID: 34341563 PMCID: PMC8610173 SCOPUS ID: 2-s2.0-85111814330 08/04/2021       34 Citations
  • Genetic therapies for the first molecular disease. (Doerfler PA, Sharma A, Porter JS, Zheng Y, Tisdale JF, Weiss MJ) J Clin Invest 2021 Apr 15;131(8) PMID: 33855970 PMCID: PMC8262557 SCOPUS ID: 2-s2.0-85104209147 04/16/2021       22 Citations
  • 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 PMID: 33846636 PMCID: PMC8192433 SCOPUS ID: 2-s2.0-85104226733 04/14/2021       366 Citations
  • 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 PMID: 31698466 PMCID: PMC6855127 SCOPUS ID: 2-s2.0-85074951547 11/08/2019       133 Citations
  • AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice. (Doyle BM, Turner SMF, Sunshine MD, Doerfler PA, Poirier AE, Vaught LA, Jorgensen ML, Falk DJ, Byrne BJ, Fuller DD) Mol Ther Methods Clin Dev 2019 Dec 13;15:194-203 PMID: 31660421 PMCID: PMC6807287 SCOPUS ID: 2-s2.0-85073432470 10/30/2019       15 Citations
  • Targeted approaches to induce immune tolerance for Pompe disease therapy. (Doerfler PA, Nayak S, Corti M, Morel L, Herzog RW, Byrne BJ) Mol Ther Methods Clin Dev 2016;3:15053 PMID: 26858964 PMCID: PMC4729315 SCOPUS ID: 2-s2.0-85007398218 02/10/2016       45 Citations
  • Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease. (Doerfler PA, Todd AG, Clément N, Falk DJ, Nayak S, Herzog RW, Byrne BJ) Hum Gene Ther 2016 Jan;27(1):43-59 PMID: 26603344 PMCID: PMC4741206 SCOPUS ID: 2-s2.0-84955262547 11/26/2015       42 Citations
  • Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice. (Su J, Sherman A, Doerfler PA, Byrne BJ, Herzog RW, Daniell H) Plant Biotechnol J 2015 Oct;13(8):1023-32 PMID: 26053072 PMCID: PMC4578979 SCOPUS ID: 2-s2.0-84942194703 06/09/2015       54 Citations
  • BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease. (Doerfler PA, Nayak S, Herzog RW, Morel L, Byrne BJ) Clin Immunol 2015 Jun;158(2):140-7 PMID: 25842186 PMCID: PMC4464966 SCOPUS ID: 2-s2.0-84928633908 04/07/2015       12 Citations
  • B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study. (Corti M, Elder M, Falk D, Lawson L, Smith B, Nayak S, Conlon T, Clément N, Erger K, Lavassani E, Green M, Doerfler P, Herzog R, Byrne B) Mol Ther Methods Clin Dev 2014;1:14033- PMID: 25541616 PMCID: PMC4275004 SCOPUS ID: 2-s2.0-84929918117 12/30/2014       110 Citations
  • Copackaging of multiple adeno-associated viral vectors in a single production step. (Doerfler PA, Byrne BJ, Clément N) Hum Gene Ther Methods 2014 Oct;25(5):269-76 PMID: 25143183 PMCID: PMC4346231 SCOPUS ID: 2-s2.0-84912075497 08/22/2014       3 Citations
  • Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent. (Nayak S, Doerfler PA, Porvasnik SL, Cloutier DD, Khanna R, Valenzano KJ, Herzog RW, Byrne BJ) PLoS One 2014;9(6):e98336 PMID: 24897114 PMCID: PMC4045583 SCOPUS ID: 2-s2.0-84902439126 06/05/2014       21 Citations
    • Last update: 06/20/2025