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Showing 1 to 8 of 8 entries
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Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells.

Molecular therapy. Methods & clinical development

Wang X, Herzog RW, Byrne BJ, Kumar SRP, Zhou Q, Buchholz CJ, Biswas M.
PMID: 28480307
Mol Ther Methods Clin Dev. 2017 Mar 29;5:76-82. doi: 10.1016/j.omtm.2017.03.005. eCollection 2017 Jun 16.

Gene-modified B cells expressing immunoglobulin G (IgG) fusion proteins have been shown to induce tolerance in several autoimmune and other disease models. However, lack of a vector suitable for gene transfer to human B cells has been an obstacle...

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Wang X, Herzog RW, Byrne BJ, et al. Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells. Mol Ther Methods Clin Dev. 2017;5:76-82doi: 10.1016/j.omtm.2017.03.005.
Wang, X., Herzog, R. W., Byrne, B. J., Kumar, S. R. P., Zhou, Q., Buchholz, C. J., & Biswas, M. (2017). Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells. Molecular therapy. Methods & clinical development, 576-82. https://doi.org/10.1016/j.omtm.2017.03.005
Wang, Xiaomei, et al. "Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells." Molecular therapy. Methods & clinical development vol. 5 (2017): 76-82. doi: https://doi.org/10.1016/j.omtm.2017.03.005
Wang X, Herzog RW, Byrne BJ, Kumar SRP, Zhou Q, Buchholz CJ, Biswas M. Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells. Mol Ther Methods Clin Dev. 2017 Mar 29;5:76-82. doi: 10.1016/j.omtm.2017.03.005. eCollection 2017 Jun 16. PMID: 28480307; PMCID: PMC5415320.
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An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8.

Molecular therapy. Methods & clinical development

Palaschak B, Marsic D, Herzog RW, Zolotukhin S, Markusic DM.
PMID: 28480313
Mol Ther Methods Clin Dev. 2017 Apr 19;5:142-152. doi: 10.1016/j.omtm.2017.04.004. eCollection 2017 Jun 16.

Multiple independent adeno-associated virus (AAV) gene therapy clinical trials for hemophilia B, utilizing different AAV serotypes, have reported a vector dose-dependent loss of circulating factor IX (FIX) protein associated with capsid-specific CD8

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Palaschak B, Marsic D, Herzog RW, et al. An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8. Mol Ther Methods Clin Dev. 2017;5:142-152doi: 10.1016/j.omtm.2017.04.004.
Palaschak, B., Marsic, D., Herzog, R. W., Zolotukhin, S., & Markusic, D. M. (2017). An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8. Molecular therapy. Methods & clinical development, 5142-152. https://doi.org/10.1016/j.omtm.2017.04.004
Palaschak, Brett, et al. "An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8." Molecular therapy. Methods & clinical development vol. 5 (2017): 142-152. doi: https://doi.org/10.1016/j.omtm.2017.04.004
Palaschak B, Marsic D, Herzog RW, Zolotukhin S, Markusic DM. An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8. Mol Ther Methods Clin Dev. 2017 Apr 19;5:142-152. doi: 10.1016/j.omtm.2017.04.004. eCollection 2017 Jun 16. PMID: 28480313; PMCID: PMC5415329.
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Engineering and .

Molecular therapy. Methods & clinical development

Biswas M, Marsic D, Li N, Zou C, Gonzalez-Aseguinolaza G, Zolotukhin I, Kumar SRP, Rana J, Butterfield JSS, Kondratov O, de Jong YP, Herzog RW, Zolotukhin S.
PMID: 33145371
Mol Ther Methods Clin Dev. 2020 Oct 04;19:347-361. doi: 10.1016/j.omtm.2020.09.019. eCollection 2020 Dec 11.

Limitations to successful gene therapy with adeno-associated virus (AAV) can comprise pre-existing neutralizing antibodies to the vector capsid that can block cellular entry, or inefficient transduction of target cells that can lead to sub-optimal expression of the therapeutic transgene....

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Biswas M, Marsic D, Li N, et al. Engineering and . Mol Ther Methods Clin Dev. 2020;19:347-361doi: 10.1016/j.omtm.2020.09.019.
Biswas, M., Marsic, D., Li, N., Zou, C., Gonzalez-Aseguinolaza, G., Zolotukhin, I., Kumar, S. R. P., Rana, J., Butterfield, J. S. S., Kondratov, O., de Jong, Y. P., Herzog, R. W., & Zolotukhin, S. (2020). Engineering and . Molecular therapy. Methods & clinical development, 19347-361. https://doi.org/10.1016/j.omtm.2020.09.019
Biswas, Moanaro, et al. "Engineering and ." Molecular therapy. Methods & clinical development vol. 19 (2020): 347-361. doi: https://doi.org/10.1016/j.omtm.2020.09.019
Biswas M, Marsic D, Li N, Zou C, Gonzalez-Aseguinolaza G, Zolotukhin I, Kumar SRP, Rana J, Butterfield JSS, Kondratov O, de Jong YP, Herzog RW, Zolotukhin S. Engineering and . Mol Ther Methods Clin Dev. 2020 Oct 04;19:347-361. doi: 10.1016/j.omtm.2020.09.019. eCollection 2020 Dec 11. PMID: 33145371; PMCID: PMC7591349.
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Dynamics of antigen presentation to transgene product-specific CD4.

Molecular therapy. Methods & clinical development

Perrin GQ, Zolotukhin I, Sherman A, Biswas M, de Jong YP, Terhorst C, Davidoff AM, Herzog RW.
PMID: 27933310
Mol Ther Methods Clin Dev. 2016 Dec 07;3:16083. doi: 10.1038/mtm.2016.83. eCollection 2016.

The tolerogenic hepatic microenvironment impedes clearance of viral infections but is an advantage in viral vector gene transfer, which often results in immune tolerance induction to transgene products. Although the underlying tolerance mechanism has been extensively studied, our understanding...

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Perrin GQ, Zolotukhin I, Sherman A, et al. Dynamics of antigen presentation to transgene product-specific CD4. Mol Ther Methods Clin Dev. 2016;3:16083doi: 10.1038/mtm.2016.83.
Perrin, G. Q., Zolotukhin, I., Sherman, A., Biswas, M., de Jong, Y. P., Terhorst, C., Davidoff, A. M., & Herzog, R. W. (2016). Dynamics of antigen presentation to transgene product-specific CD4. Molecular therapy. Methods & clinical development, 316083. https://doi.org/10.1038/mtm.2016.83
Perrin, George Q, et al. "Dynamics of antigen presentation to transgene product-specific CD4." Molecular therapy. Methods & clinical development vol. 3 (2016): 16083. doi: https://doi.org/10.1038/mtm.2016.83
Perrin GQ, Zolotukhin I, Sherman A, Biswas M, de Jong YP, Terhorst C, Davidoff AM, Herzog RW. Dynamics of antigen presentation to transgene product-specific CD4. Mol Ther Methods Clin Dev. 2016 Dec 07;3:16083. doi: 10.1038/mtm.2016.83. eCollection 2016. PMID: 27933310; PMCID: PMC5142511.
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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.

Nature communications

Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen SJ, Herzog RW, Duan D.
PMID: 34819506
Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7.

Adeno-associated virus (AAV)-mediated CRISPR-Cas9 editing holds promise to treat many diseases. The immune response to bacterial-derived Cas9 has been speculated as a hurdle for AAV-CRISPR therapy. However, immunological consequences of AAV-mediated Cas9 expression have thus far not been thoroughly...

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Hakim CH, Kumar SRP, Pérez-López DO, et al. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nat Commun. 2021;12(1):6769doi: 10.1038/s41467-021-26830-7.
Hakim, C. H., Kumar, S. R. P., Pérez-López, D. O., Wasala, N. B., Zhang, D., Yue, Y., Teixeira, J., Pan, X., Zhang, K., Million, E. D., Nelson, C. E., Metzger, S., Han, J., Louderman, J. A., Schmidt, F., Feng, F., Grimm, D., Smith, B. F., Yao, G., Yang, N. N., Gersbach, C. A., Chen, S. J., Herzog, R. W., & Duan, D. (2021). Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nature communications, 12(1), 6769. https://doi.org/10.1038/s41467-021-26830-7
Hakim, Chady H, et al. "Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models." Nature communications vol. 12,1 (2021): 6769. doi: https://doi.org/10.1038/s41467-021-26830-7
Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen SJ, Herzog RW, Duan D. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7. PMID: 34819506; PMCID: PMC8613397.
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Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A.

Frontiers in immunology

Sherman A, Biswas M, Herzog RW.
PMID: 29225598
Front Immunol. 2017 Nov 24;8:1604. doi: 10.3389/fimmu.2017.01604. eCollection 2017.

Hemophilia A (coagulation factor VIII deficiency) is a debilitating genetic disorder that is primarily treated with intravenous replacement therapy. Despite a variety of factor VIII protein formulations available, the risk of developing anti-dug antibodies ("inhibitors") remains. Overall, 20-30% of...

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Sherman A, Biswas M, Herzog RW. Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A. Front Immunol. 2017;8:1604doi: 10.3389/fimmu.2017.01604.
Sherman, A., Biswas, M., & Herzog, R. W. (2017). Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A. Frontiers in immunology, 81604. https://doi.org/10.3389/fimmu.2017.01604
Sherman, Alexandra, et al. "Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A." Frontiers in immunology vol. 8 (2017): 1604. doi: https://doi.org/10.3389/fimmu.2017.01604
Sherman A, Biswas M, Herzog RW. Innovative Approaches for Immune Tolerance to Factor VIII in the Treatment of Hemophilia A. Front Immunol. 2017 Nov 24;8:1604. doi: 10.3389/fimmu.2017.01604. eCollection 2017. PMID: 29225598; PMCID: PMC5705551.
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Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice.

Molecular therapy. Methods & clinical development

Zou C, Vercauteren KOA, Michailidis E, Kabbani M, Zoluthkin I, Quirk C, Chiriboga L, Yazicioglu M, Anguela XM, Meuleman P, High KA, Herzog RW, de Jong YP.
PMID: 32637450
Mol Ther Methods Clin Dev. 2020 Jun 02;18:189-198. doi: 10.1016/j.omtm.2020.05.033. eCollection 2020 Sep 11.

Adeno-associated virus (AAV) vector serotypes vary in their ability to transduce hepatocytes from different species. Chimeric mouse models harboring human hepatocytes have shown translational promise for liver-directed gene therapies. However, many variables that influence human hepatocyte transduction and transgene...

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Zou C, Vercauteren KOA, Michailidis E, et al. Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice. Mol Ther Methods Clin Dev. 2020;18:189-198doi: 10.1016/j.omtm.2020.05.033.
Zou, C., Vercauteren, K. O. A., Michailidis, E., Kabbani, M., Zoluthkin, I., Quirk, C., Chiriboga, L., Yazicioglu, M., Anguela, X. M., Meuleman, P., High, K. A., Herzog, R. W., & de Jong, Y. P. (2020). Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice. Molecular therapy. Methods & clinical development, 18189-198. https://doi.org/10.1016/j.omtm.2020.05.033
Zou, Chenhui, et al. "Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice." Molecular therapy. Methods & clinical development vol. 18 (2020): 189-198. doi: https://doi.org/10.1016/j.omtm.2020.05.033
Zou C, Vercauteren KOA, Michailidis E, Kabbani M, Zoluthkin I, Quirk C, Chiriboga L, Yazicioglu M, Anguela XM, Meuleman P, High KA, Herzog RW, de Jong YP. Experimental Variables that Affect Human Hepatocyte AAV Transduction in Liver Chimeric Mice. Mol Ther Methods Clin Dev. 2020 Jun 02;18:189-198. doi: 10.1016/j.omtm.2020.05.033. eCollection 2020 Sep 11. PMID: 32637450; PMCID: PMC7326722.
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Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models.

Nature communications

Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen SJ, Herzog RW, Duan D.
PMID: 34819506
Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7.

Adeno-associated virus (AAV)-mediated CRISPR-Cas9 editing holds promise to treat many diseases. The immune response to bacterial-derived Cas9 has been speculated as a hurdle for AAV-CRISPR therapy. However, immunological consequences of AAV-mediated Cas9 expression have thus far not been thoroughly...

Cite
Hakim CH, Kumar SRP, Pérez-López DO, et al. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nat Commun. 2021;12(1):6769doi: 10.1038/s41467-021-26830-7.
Hakim, C. H., Kumar, S. R. P., Pérez-López, D. O., Wasala, N. B., Zhang, D., Yue, Y., Teixeira, J., Pan, X., Zhang, K., Million, E. D., Nelson, C. E., Metzger, S., Han, J., Louderman, J. A., Schmidt, F., Feng, F., Grimm, D., Smith, B. F., Yao, G., Yang, N. N., Gersbach, C. A., Chen, S. J., Herzog, R. W., & Duan, D. (2021). Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nature communications, 12(1), 6769. https://doi.org/10.1038/s41467-021-26830-7
Hakim, Chady H, et al. "Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models." Nature communications vol. 12,1 (2021): 6769. doi: https://doi.org/10.1038/s41467-021-26830-7
Hakim CH, Kumar SRP, Pérez-López DO, Wasala NB, Zhang D, Yue Y, Teixeira J, Pan X, Zhang K, Million ED, Nelson CE, Metzger S, Han J, Louderman JA, Schmidt F, Feng F, Grimm D, Smith BF, Yao G, Yang NN, Gersbach CA, Chen SJ, Herzog RW, Duan D. Cas9-specific immune responses compromise local and systemic AAV CRISPR therapy in multiple dystrophic canine models. Nat Commun. 2021 Nov 24;12(1):6769. doi: 10.1038/s41467-021-26830-7. PMID: 34819506; PMCID: PMC8613397.
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Showing 1 to 8 of 8 entries