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The national blueprint for future basic and translational research to understand factor VIII immunogenicity: NHLBI State of the Science Workshop on factor VIII inhibitors.

Meeks SL, Herzog RW; Members of Working Group 3, the NHLBI State of the Science Workshop on factor VIII inhibitors: Generating a national blueprint for future research.

Haemophilia. 2019 Jul;25(4):595-602. doi: 10.1111/hae.13740.


SLAMF6 as a Regulator of Exhausted CD8+ T Cells in Cancer.

Yigit B, Wang N, Ten Hacken E, Chen SS, Bhan AK, Suarez-Fueyo A, Katsuyama E, Tsokos GC, Chiorazzi N, Wu CJ, Burger JA, Herzog RW, Engel P, Terhorst C.

Cancer Immunol Res. 2019 Sep;7(9):1485-1496. doi: 10.1158/2326-6066.CIR-18-0664. Epub 2019 Jul 17.


Plant cell-made protein antigens for induction of Oral tolerance.

Daniell H, Kulis M, Herzog RW.

Biotechnol Adv. 2019 Jun 26. pii: S0734-9750(19)30103-X. doi: 10.1016/j.biotechadv.2019.06.012. [Epub ahead of print] Review.


TLR9-Activating CpG-B ODN but Not TLR7 Agonists Triggers Antibody Formation to Factor IX in Muscle Gene Transfer.

Butterfield JSS, Biswas M, Shirley JL, Kumar SRP, Sherman A, Terhorst C, Ling C, Herzog RW.

Hum Gene Ther Methods. 2019 Jun;30(3):81-92. doi: 10.1089/hgtb.2019.013.


The Checkpoint Regulator SLAMF3 Preferentially Prevents Expansion of Auto-Reactive B Cells Generated by Graft-vs.-Host Disease.

Wang N, Yigit B, van der Poel CE, Cuenca M, Carroll MC, Herzog RW, Engel P, Terhorst C.

Front Immunol. 2019 Apr 17;10:831. doi: 10.3389/fimmu.2019.00831. eCollection 2019.


Reprogrammed CD4+ T Cells That Express FoxP3+ Control Inhibitory Antibody Formation in Hemophilia A Mice.

Herzog RW, Kuteyeva V, Saboungi R, Terhorst C, Biswas M.

Front Immunol. 2019 Feb 20;10:274. doi: 10.3389/fimmu.2019.00274. eCollection 2019.


AAV-Mediated Gene Delivery to the Liver: Overview of Current Technologies and Methods.

Palaschak B, Herzog RW, Markusic DM.

Methods Mol Biol. 2019;1950:333-360. doi: 10.1007/978-1-4939-9139-6_20. Review.


Update on clinical gene therapy for hemophilia.

Perrin GQ, Herzog RW, Markusic DM.

Blood. 2019 Jan 31;133(5):407-414. doi: 10.1182/blood-2018-07-820720. Epub 2018 Dec 17. Review.


AAV Immunogenicity: New Answers Create New Questions.

Shirley JL, Herzog RW.

Mol Ther. 2018 Nov 7;26(11):2538-2539. doi: 10.1016/j.ymthe.2018.10.004. Epub 2018 Oct 23. No abstract available.


SLAMF6 in health and disease: Implications for therapeutic targeting.

Yigit B, Wang N, Herzog RW, Terhorst C.

Clin Immunol. 2019 Jul;204:3-13. doi: 10.1016/j.clim.2018.10.013. Epub 2018 Oct 23. Review. No abstract available.


Tolerance induction in hemophilia: innovation and accomplishments.

Sherman A, Biswas M, Herzog RW.

Curr Opin Hematol. 2018 Sep;25(5):365-372. doi: 10.1097/MOH.0000000000000446. Review.


Impact of neutralizing antibodies against AAV is a key consideration in gene transfer to nonhuman primates.

Xiao W, Gao G, Ling C, Herzog RW, Xiao X, Samulski RJ.

Nat Med. 2018 Jun;24(6):699. doi: 10.1038/s41591-018-0062-2. No abstract available.


Gene Therapy With Regulatory T Cells: A Beneficial Alliance.

Biswas M, Kumar SRP, Terhorst C, Herzog RW.

Front Immunol. 2018 Mar 19;9:554. doi: 10.3389/fimmu.2018.00554. eCollection 2018. Review.


Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A.

Sherman A, Biswas M, Herzog RW.

Front Immunol. 2017 Nov 24;8:1604. doi: 10.3389/fimmu.2017.01604. eCollection 2017. Review.


Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease.

Corti M, Liberati C, Smith BK, Lawson LA, Tuna IS, Conlon TJ, Coleman KE, Islam S, Herzog RW, Fuller DD, Collins SW, Byrne BJ.

Hum Gene Ther Clin Dev. 2017 Dec;28(4):208-218. doi: 10.1089/humc.2017.146.


Expression and assembly of largest foreign protein in chloroplasts: oral delivery of human FVIII made in lettuce chloroplasts robustly suppresses inhibitor formation in haemophilia A mice.

Kwon KC, Sherman A, Chang WJ, Kamesh A, Biswas M, Herzog RW, Daniell H.

Plant Biotechnol J. 2018 Jun;16(6):1148-1160. doi: 10.1111/pbi.12859. Epub 2017 Dec 7.


Regulatory T cells and TLR9 activation shape antibody formation to a secreted transgene product in AAV muscle gene transfer.

Herzog RW, Cooper M, Perrin GQ, Biswas M, Martino AT, Morel L, Terhorst C, Hoffman BE.

Cell Immunol. 2019 Aug;342:103682. doi: 10.1016/j.cellimm.2017.07.012. Epub 2017 Aug 1.


Complexity of immune responses to AAV transgene products - Example of factor IX.

Herzog RW.

Cell Immunol. 2019 Aug;342:103658. doi: 10.1016/j.cellimm.2017.05.006. Epub 2017 May 29. Review.


An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8+ T Cells.

Palaschak B, Marsic D, Herzog RW, Zolotukhin S, Markusic DM.

Mol Ther Methods Clin Dev. 2017 Apr 19;5:142-152. doi: 10.1016/j.omtm.2017.04.004. eCollection 2017 Jun 16.


Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells.

Wang X, Herzog RW, Byrne BJ, Kumar SRP, Zhou Q, Buchholz CJ, Biswas M.

Mol Ther Methods Clin Dev. 2017 Mar 29;5:76-82. doi: 10.1016/j.omtm.2017.03.005. eCollection 2017 Jun 16.


Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells.

Rogers GL, Shirley JL, Zolotukhin I, Kumar SRP, Sherman A, Perrin GQ, Hoffman BE, Srivastava A, Basner-Tschakarjan E, Wallet MA, Terhorst C, Biswas M, Herzog RW.

Blood. 2017 Jun 15;129(24):3184-3195. doi: 10.1182/blood-2016-11-751040. Epub 2017 May 3.


Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.

Markusic DM, Nichols TC, Merricks EP, Palaschak B, Zolotukhin I, Marsic D, Zolotukhin S, Srivastava A, Herzog RW.

J Transl Med. 2017 May 1;15(1):94. doi: 10.1186/s12967-017-1200-1.


The Balance between CD8+ T Cell-Mediated Clearance of AAV-Encoded Antigen in the Liver and Tolerance Is Dependent on the Vector Dose.

Kumar SRP, Hoffman BE, Terhorst C, de Jong YP, Herzog RW.

Mol Ther. 2017 Apr 5;25(4):880-891. doi: 10.1016/j.ymthe.2017.02.014. Epub 2017 Mar 9.


Oral Tolerance Induction in Hemophilia B Dogs Fed with Transplastomic Lettuce.

Herzog RW, Nichols TC, Su J, Zhang B, Sherman A, Merricks EP, Raymer R, Perrin GQ, Häger M, Wiinberg B, Daniell H.

Mol Ther. 2017 Feb 1;25(2):512-522. doi: 10.1016/j.ymthe.2016.11.009.


Driving the hemophilia tolerance CAR.

Herzog RW.

Blood. 2017 Jan 12;129(2):142-144. doi: 10.1182/blood-2016-11-753160. No abstract available.


Dynamics of antigen presentation to transgene product-specific CD4+ T cells and of Treg induction upon hepatic AAV gene transfer.

Perrin GQ, Zolotukhin I, Sherman A, Biswas M, de Jong YP, Terhorst C, Davidoff AM, Herzog RW.

Mol Ther Methods Clin Dev. 2016 Dec 7;3:16083. eCollection 2016.


Potential for cellular stress response to hepatic factor VIII expression from AAV vector.

Zolotukhin I, Markusic DM, Palaschak B, Hoffman BE, Srikanthan MA, Herzog RW.

Mol Ther Methods Clin Dev. 2016 Sep 28;3:16063. eCollection 2016.


A Cure For Hemophilia: the Promise Becomes a Reality.

Herzog RW.

Mol Ther. 2016 Sep;24(9):1503-4. doi: 10.1038/mt.2016.169. No abstract available.


Combination therapy for inhibitor reversal in haemophilia A using monoclonal anti-CD20 and rapamycin.

Biswas M, Rogers GL, Sherman A, Byrne BJ, Markusic DM, Jiang H, Herzog RW.

Thromb Haemost. 2017 Jan 5;117(1):33-43. doi: 10.1160/TH16-05-0404. Epub 2016 Sep 29.


Innovating immune tolerance induction for haemophilia.

Batsuli G, Meeks SL, Herzog RW, Lacroix-Desmazes S.

Haemophilia. 2016 Jul;22 Suppl 5:31-5. doi: 10.1111/hae.12989. Review.


Clinical development of gene therapy: results and lessons from recent successes.

Kumar SR, Markusic DM, Biswas M, High KA, Herzog RW.

Mol Ther Methods Clin Dev. 2016 May 25;3:16034. doi: 10.1038/mtm.2016.34. eCollection 2016. Review.


Expanded Opportunities for Methods and Clinical Development.

Herzog RW.

Mol Ther. 2016 Apr;24(4):655-6. doi: 10.1038/mt.2016.44. No abstract available.


Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.

Vercauteren K, Hoffman BE, Zolotukhin I, Keeler GD, Xiao JW, Basner-Tschakarjan E, High KA, Ertl HC, Rice CM, Srivastava A, de Jong YP, Herzog RW.

Mol Ther. 2016 Jun;24(6):1042-1049. doi: 10.1038/mt.2016.61. Epub 2016 Mar 29.


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 Jan 27;3:15053. doi: 10.1038/mtm.2015.53. eCollection 2016. Review.


Low cost delivery of proteins bioencapsulated in plant cells to human non-immune or immune modulatory cells.

Xiao Y, Kwon KC, Hoffman BE, Kamesh A, Jones NT, Herzog RW, Daniell H.

Biomaterials. 2016 Feb;80:68-79. doi: 10.1016/j.biomaterials.2015.11.051. Epub 2015 Dec 2.


Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A.

Hum Gene Ther. 2015 Dec;26(12):779-81. doi: 10.1089/hum.2015.29014.kib. No abstract available.


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. doi: 10.1089/hum.2015.103.


In vivo induction of regulatory T cells for immune tolerance in hemophilia.

Wang X, Terhorst C, Herzog RW.

Cell Immunol. 2016 Mar;301:18-29. doi: 10.1016/j.cellimm.2015.10.001. Epub 2015 Oct 9. Review.


Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning.

Corti M, Cleaver B, Clément N, Conlon TJ, Faris KJ, Wang G, Benson J, Tarantal AF, Fuller D, Herzog RW, Byrne BJ.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):185-93. doi: 10.1089/humc.2015.068.


Hemophilia Gene Therapy: Caught Between a Cure and an Immune Response.

Herzog RW.

Mol Ther. 2015 Sep;23(9):1411-2. doi: 10.1038/mt.2015.135. No abstract available.


Low cost industrial production of coagulation factor IX bioencapsulated in lettuce cells for oral tolerance induction in hemophilia B.

Su J, Zhu L, Sherman A, Wang X, Lin S, Kamesh A, Norikane JH, Streatfield SJ, Herzog RW, Daniell H.

Biomaterials. 2015 Nov;70:84-93. doi: 10.1016/j.biomaterials.2015.08.004. Epub 2015 Aug 5.


Treg: tolerance vs immunity.

Biswas M, Terhorst C, Herzog RW.

Oncotarget. 2015 Aug 21;6(24):19956-7. No abstract available.


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. doi: 10.1111/pbi.12413. Epub 2015 Jun 5.


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. doi: 10.1016/j.clim.2015.03.022. Epub 2015 Apr 1.


Synergy between rapamycin and FLT3 ligand enhances plasmacytoid dendritic cell-dependent induction of CD4+CD25+FoxP3+ Treg.

Biswas M, Sarkar D, Kumar SR, Nayak S, Rogers GL, Markusic DM, Liao G, Terhorst C, Herzog RW.

Blood. 2015 May 7;125(19):2937-47. doi: 10.1182/blood-2014-09-599266. Epub 2015 Apr 1.


Molecular therapy: at the cutting edge of methodology and clinical development.

Herzog RW.

Mol Ther. 2015 Mar;23(3):409-10. doi: 10.1038/mt.2015.23. No abstract available.


Plant-based oral tolerance to hemophilia therapy employs a complex immune regulatory response including LAP+CD4+ T cells.

Wang X, Su J, Sherman A, Rogers GL, Liao G, Hoffman BE, Leong KW, Terhorst C, Daniell H, Herzog RW.

Blood. 2015 Apr 9;125(15):2418-27. doi: 10.1182/blood-2014-08-597070. Epub 2015 Feb 19.


Unique Roles of TLR9- and MyD88-Dependent and -Independent Pathways in Adaptive Immune Responses to AAV-Mediated Gene Transfer.

Rogers GL, Suzuki M, Zolotukhin I, Markusic DM, Morel LM, Lee B, Ertl HC, Herzog RW.

J Innate Immun. 2015;7(3):302-14. doi: 10.1159/000369273. Epub 2015 Jan 20.


Development of Gene Transfer for Induction of Antigen-specific Tolerance.

Sack BK, Herzog RW, Terhorst C, Markusic DM.

Mol Ther Methods Clin Dev. 2014 Apr 30;1:14013.


Gene therapy for hemophilia.

Rogers GL, Herzog RW.

Front Biosci (Landmark Ed). 2015 Jan 1;20:556-603. Review.

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