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ArticlesPeer ReviewedFull Text
Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice
Author / Creator:Zhou, Yuan ; Zhang, Chen ; Xiao, Weidong ; Herzog, Roland W. ; Han, Renzhi
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2024-07, Vol.15 (1), p.6141-14, Article 6141
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Hemophilia Gene Therapy: Caught Between a Cure and an Immune Response
Author / Creator:Herzog, Roland W.
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2015-09, Vol.23 (9), p.1411-1412
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A Cure For Hemophilia: the Promise Becomes a Reality
Author / Creator:Herzog, Roland W
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2016-09, Vol.24 (9), p.1503-1504
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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models
Author / Creator:Hakim, Chady H. ; Kumar, Sandeep R. P. ; Pérez-López, Dennis O. ; Wasala, Nalinda B. ; Zhang, Dong ; Yue, Yongping ; Teixeira, James ; Pan, Xiufang ; Zhang, Keqing ; Million, Emily D. ; Nelson, Christopher E. ; Metzger, Samantha ; Han, Jin ; Louderman, Jacqueline A. ; Schmidt, Florian ; Feng, Feng ; Grimm, Dirk ; Smith, Bruce F. ; Yao, Gang ; Yang, N. Nora ; Gersbach, Charles A. ; Chen, Shi-jie ; Herzog, Roland W. ; Duan, Dongsheng
Publisher:London: Nature Publishing Group UK
Journal title:Nature communications, 2021-11, Vol.12 (1), p.6769-12, Article 6769
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Adeno‐Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines
Author / Creator:Burke, Matthew J. ; Blatt, Braiden M. ; Teixeira, James A. ; Pérez‐López, Dennis O. ; Yue, Yongping ; Pan, Xiufang ; Hakim, Chady H. ; Yao, Gang ; Herzog, Roland W. ; Duan, Dongsheng
Publisher:Germany: John Wiley & Sons, Inc
Journal title:Journal of cachexia, sarcopenia and muscle, 2025-02, Vol.16 (1), p.e13681-n/a
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Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid
Author / Creator:Vercauteren, Koen ; Hoffman, Brad E ; Zolotukhin, Irene ; Keeler, Geoffrey D ; Xiao, Jing W ; Basner-Tschakarjan, Etiena ; High, Katherine A ; Ertl, Hildegund CJ ; Rice, Charles M ; Srivastava, Arun ; de Jong, Ype P ; Herzog, Roland W
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2016-06, Vol.24 (6), p.1042-1049
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Clinical development of gene therapy: results and lessons from recent successes
Author / Creator:Kumar, Sandeep Rp ; Markusic, David M ; Biswas, Moanaro ; High, Katherine A ; Herzog, Roland W
Publisher:United States: Elsevier Limited
Journal title:Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16034-16034, Article 16034
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Expansion, in vivo–ex vivo cycling, and genetic manipulation of primary human hepatocytes
Author / Creator:Michailidis, Eleftherios ; Vercauteren, Koen ; Mancio-Silva, Liliana ; Andrus, Linda ; Jahan, Cyprien ; Ricardo-Lax, Inna ; Zou, Chenhui ; Kabbani, Mohammad ; Park, Paul ; Quirk, Corrine ; Pyrgaki, Christina ; Razooky, Brandon ; Verhoye, Lieven ; Zoluthkin, Irene ; Lu, Wei-Yu ; Forbes, Stuart J. ; Chiriboga, Luis ; Theise, Neil D. ; Herzog, Roland W. ; Suemizu, Hiroshi ; Schneider, William M. ; Shlomai, Amir ; Meuleman, Philip ; Bhatia, Sangeeta N. ; Rice, Charles M. ; de Jong, Ype P.
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2020-01, Vol.117 (3), p.1678-1688
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Chemical modification of AAV9 capsid with N-ethyl maleimide alters vector tissue tropism
Author / Creator:Mulcrone, Patrick L. ; Lam, Anh K. ; Frabutt, Dylan ; Zhang, Junping ; Chrzanowski, Matthew ; Herzog, Roland W. ; Xiao, Weidong
Publisher:London: Nature Publishing Group UK
Journal title:Scientific reports, 2023-05, Vol.13 (1), p.8436-14, Article 8436
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Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
Author / Creator:Zhong, Li ; Li, Baozheng ; Mah, Cathryn S ; Govindasamy, Lakshmanan ; Agbandje-McKenna, Mavis ; Cooper, Mario ; Herzog, Roland W ; Zolotukhin, Irene ; Warrington, Kenneth H. Jr ; Weigel-Van Aken, Kirsten A ; Hobbs, Jacqueline A ; Zolotukhin, Sergei ; Muzyczka, Nicholas ; Srivastava, Arun
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2008-06, Vol.105 (22), p.7827-7832
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Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies
Author / Creator:Markusic, David M. ; Hoffman, Brad E. ; Perrin, George Q. ; Nayak, Sushrusha ; Wang, Xiaomei ; LoDuca, Paul A. ; High, Katherine A. ; Herzog, Roland W.
Publisher:London: Nature Publishing Group UK
Journal title:EMBO molecular medicine, 2013-11, Vol.5 (11), p.1698-1709
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Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice
Author / Creator:Verma, Dheeraj ; Moghimi, Babak ; LoDuca, Paul A ; Singh, Harminder D ; Hoffman, Brad E ; Herzog, Roland W ; Daniell, Henry
Publisher:United States: National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2010-04, Vol.107 (15), p.7101-7106
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Liver-specific in vivo base editing of Angptl3 via AAV delivery efficiently lowers blood lipid levels in mice
Author / Creator:Zuo, Yuanbojiao ; Zhang, Chen ; Zhou, Yuan ; Li, Haiwen ; Xiao, Weidong ; Herzog, Roland W ; Xu, Jie ; Zhang, Jifeng ; Chen, Y Eugene ; Han, Renzhi
Publisher:England: BioMed Central Ltd
Journal title:Cell & bioscience, 2023-06, Vol.13 (1), p.109-109, Article 109
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Viral Vector Based Immunotherapy for Peanut Allergy
Author / Creator:Gonzalez-Visiedo, Miguel ; Herzog, Roland W ; Munoz-Melero, Maite ; Blessinger, Sophia A ; Cook-Mills, Joan M ; Daniell, Henry ; Markusic, David M
Publisher:Switzerland: MDPI AG
Journal title:Viruses, 2024-07, Vol.16 (7), p.1125
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Redundancy in Innate Immune Pathways That Promote CD8 + T-Cell Responses in AAV1 Muscle Gene Transfer
Author / Creator:Li, Ning ; Kumar, Sandeep R P ; Cao, Di ; Munoz-Melero, Maite ; Arisa, Sreevani ; Brian, Bridget A ; Greenwood, Calista M ; Yamada, Kentaro ; Duan, Dongsheng ; Herzog, Roland W
Publisher:Switzerland: MDPI AG
Journal title:Viruses, 2024-10, Vol.16 (10), p.1507
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High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines
Author / Creator:Markusic, David M ; Herzog, Roland W ; Aslanidi, George V ; Hoffman, Brad E ; Li, Baozheng ; Li, Mengxin ; Jayandharan, Giridhara R. ; Ling, Chen ; Zolotukhin, Irene ; Ma, Wenqin ; Zolotukhin, Sergei ; Srivastava, Arun ; Zhong, Li
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2010-12, Vol.18 (12), p.2048-2056
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Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy
Author / Creator:Sack, Brandon K ; Merchant, Sherin ; Markusic, David M ; Nathwani, Amit C ; Davidoff, Andrew M ; Byrne, Barry J ; Herzog, Roland W
Publisher:United States: Public Library of Science
Journal title:PloS one, 2012-05, Vol.7 (5), p.e37671-e37671
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Immune Tolerance Induction to Factor IX through B Cell Gene Transfer: TLR9 Signaling Delineates between Tolerogenic and Immunogenic B Cells
Author / Creator:Wang, Xiaomei ; Moghimi, Babak ; Zolotukhin, Irene ; Morel, Laurence M ; Cao, Ou ; Herzog, Roland W
Publisher:United States: Elsevier Inc
Journal title:Molecular therapy, 2014-06, Vol.22 (6), p.1139-1150
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Unique Roles of TLR9- and MyD88-Dependent and -Independent Pathways in Adaptive Immune Responses to AAV-Mediated Gene Transfer
Author / Creator:Rogers, Geoffrey L. ; Suzuki, Masataka ; Zolotukhin, Irene ; Markusic, David M. ; Morel, Laurence M. ; Lee, Brendan ; Ertl, Hildegund C.J. ; Herzog, Roland W.
Publisher:Basel, Switzerland: S. Karger AG
Journal title:Journal of innate immunity, 2015-01, Vol.7 (3), p.302-314
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Activation of the NF-κB pathway by adeno-associated virus (AAV) vectors and its implications in immune response and gene therapy
Author / Creator:Jayandharan, Giridhara R ; Aslanidi, George ; Martino, Ashley T ; Jahn, Stephan C ; Perrin, George Q ; Herzog, Roland W ; Srivastava, Arun
Publisher:National Academy of Sciences
Journal title:Proceedings of the National Academy of Sciences - PNAS, 2011-03, Vol.108 (9), p.3743-3748
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Showing 1 to 20 of 88 results

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Systemic delivery of full-length dystrophin in Duchenne muscular dystrophy mice

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

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A Cure For Hemophilia: the Promise Becomes a Reality

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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models

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Adeno‐Associated Virus 8 and 9 Myofibre Type/Size Tropism Profiling Reveals Therapeutic Effect of Microdystrophin in Canines

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Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

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

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Expansion, in vivo–ex vivo cycling, and genetic manipulation of primary human hepatocytes

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Chemical modification of AAV9 capsid with N-ethyl maleimide alters vector tissue tropism

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Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

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Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

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Oral delivery of bioencapsulated coagulation factor IX prevents inhibitor formation and fatal anaphylaxis in hemophilia B mice

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Liver-specific in vivo base editing of Angptl3 via AAV delivery efficiently lowers blood lipid levels in mice

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Viral Vector Based Immunotherapy for Peanut Allergy

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Redundancy in Innate Immune Pathways That Promote CD8 + T-Cell Responses in AAV1 Muscle Gene Transfer

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High-efficiency Transduction and Correction of Murine Hemophilia B Using AAV2 Vectors Devoid of Multiple Surface-exposed Tyrosines

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Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy

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Immune Tolerance Induction to Factor IX through B Cell Gene Transfer: TLR9 Signaling Delineates between Tolerogenic and Immunogenic B Cells

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

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Activation of the NF-κB pathway by adeno-associated virus (AAV) vectors and its implications in immune response and gene therapy

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