| Chromothripsis as an on-target consequence of CRISPR–Cas9 genome editing ML Leibowitz, S Papathanasiou, PA Doerfler, LJ Blaine, L Sun, Y Yao, ... Nature genetics 53 (6), 895-905, 2021 | 406 | 2021 |
| Genome editing of HBG1 and HBG2 to induce fetal hemoglobin JY Métais, PA Doerfler, T Mayuranathan, DE Bauer, SC Fowler, MM Hsieh, ... Blood advances 3 (21), 3379-3392, 2019 | 141 | 2019 |
| B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study M Corti, ME Elder, DJ Falk, L Lawson, BK Smith, S Nayak, TJ Conlon, ... Molecular Therapy-Methods & Clinical Development 1, 2014 | 123 | 2014 |
| Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice J Su, A Sherman, PA Doerfler, BJ Byrne, RW Herzog, H Daniell Plant biotechnology journal 13 (8), 1023-1032, 2015 | 65 | 2015 |
| Copackaged AAV9 vectors promote simultaneous immune tolerance and phenotypic correction of Pompe disease PA Doerfler, AG Todd, N Clement, DJ Falk, S Nayak, RW Herzog, ... Human gene therapy 27 (1), 43-59, 2016 | 59 | 2016 |
| Targeted approaches to induce immune tolerance for Pompe disease therapy PA Doerfler, S Nayak, M Corti, L Morel, RW Herzog, BJ Byrne Molecular Therapy Methods & Clinical Development 3, 2016 | 49 | 2016 |
| Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin PA Doerfler, R Feng, Y Li, LE Palmer, SN Porter, HW Bell, M Crossley, ... Nature genetics 53 (8), 1177-1186, 2021 | 28 | 2021 |
| Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent S Nayak, PA Doerfler, SL Porvasnik, DD Cloutier, R Khanna, ... PLoS One 9 (6), e98336, 2014 | 28 | 2014 |
| Potent and uniform fetal hemoglobin induction via base editing T Mayuranathan, GA Newby, R Feng, Y Yao, KD Mayberry, CR Lazzarotto, ... Nature genetics 55 (7), 1210-1220, 2023 | 22 | 2023 |
| Genetic therapies for the first molecular disease PA Doerfler, A Sharma, JS Porter, Y Zheng, JF Tisdale, MJ Weiss The Journal of Clinical Investigation 131 (8), 2021 | 21 | 2021 |
| Activation of γ-globin expression by hypoxia-inducible factor 1α R Feng, T Mayuranathan, P Huang, PA Doerfler, Y Li, Y Yao, J Zhang, ... Nature 610 (7933), 783-790, 2022 | 20 | 2022 |
| Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells K Qin, P Huang, R Feng, CA Keller, SA Peslak, E Khandros, MS Saari, ... Nature genetics 54 (6), 874-884, 2022 | 20 | 2022 |
| Methods of packaging multiple adeno-associated virus vectors BJ Byrne, PA Doerfler, N Clement US Patent 10,781,459, 2020 | 18 | 2020 |
| AAV gene therapy utilizing glycosylation-independent lysosomal targeting tagged GAA in the hypoglossal motor system of pompe mice BM Doyle, SMF Turner, MD Sunshine, PA Doerfler, AE Poirier, LA Vaught, ... Molecular Therapy-Methods & Clinical Development 15, 194-203, 2019 | 16 | 2019 |
| BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease PA Doerfler, S Nayak, RW Herzog, L Morel, BJ Byrne Clinical Immunology 158 (2), 140-147, 2015 | 16 | 2015 |
| Limitations of mouse models for sickle cell disease conferred by their human globin transgene configurations KJ Woodard, PA Doerfler, KD Mayberry, A Sharma, R Levine, J Yen, ... Disease Models & Mechanisms 15 (6), dmm049463, 2022 | 9 | 2022 |
| Copackaging of multiple adeno-associated viral vectors in a single production step PA Doerfler, BJ Byrne, N Clément Human Gene Therapy Methods 25 (5), 269-276, 2014 | 7 | 2014 |
| Regulation of fetal hemoglobin expression by the VHL-HIF1α oxygen sensing system R Feng, T Mayuranathan, P Huang, PA Doerfler, Y Li, Y Yao, J Zhang, ... Blood 138, 574, 2021 | 4 | 2021 |
| Pharmacological chaperones prevent the precipitation of rhGAA by anti-GAA antibodies during enzyme replacement therapy PA Doerfler, JS Kelley, S Nayak, LA Lawson, BJ Byrne Molecular Genetics and Metabolism 2 (111), S38, 2014 | 2 | 2014 |
| CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies JY Metais, PA Doerfler, T Mayuranathan, DE Bauer, S Fowler, M Hsieh, ... Blood 134, 2066, 2019 | 1 | 2019 |
Barry J. ByrneUniversity of Florida在 ufl.edu 的电子邮件经过验证
