Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver von Mingozzi, Federico, Hasbrouck, Nicole C., Basner-Tschakarjan, Etiena, Edmonson, Shyrie A., Hui, Daniel J., Sabatino, Denise E., Zhou, Shangzhen, Wright, J. Fraser, Jiang, Haiyan, Pierce, Glenn F., Arruda, Valder R., High, Katherine A.

Volltext

Distinct Induction Patterns and Functions of Two Closely Related Interferon-inducible Human Genes, ISG54 and ISG56 von Terenzi, Fulvia, Hui, Daniel J., Merrick, William C., Sen, Ganes C.

Volltext

Diverse IgG subclass responses to adeno-associated virus infection and vector administration von Murphy, Samuel L, Li, Hojun, Mingozzi, Federico, Sabatino, Denise E, Hui, Daniel J, Edmonson, Shyrie A, High, Katherine A

Volltext

Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs von Haurigot, Virginia, Mingozzi, Federico, Buchlis, George, Hui, Daniel J, Chen, Yifeng, Basner-Tschakarjan, Etiena, Arruda, Valder R, Radu, Antoneta, Franck, Helen G, Wright, J Fraser, Zhou, Shangzhen, Stedman, Hansell H, Bellinger, Dwight A, Nichols, Timothy C, High, Katherine A

Volltext

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes von Hui, Daniel J, Edmonson, Shyrie C, Podsakoff, Gregory M, Pien, Gary C, Ivanciu, Lacramioara, Camire, Rodney M, Ertl, Hildegund, Mingozzi, Federico, High, Katherine A, Basner-Tschakarjan, Etiena

Volltext

Mouse p56 Blocks a Distinct Function of Eukaryotic Initiation Factor 3 in Translation Initiation von Hui, Daniel J., Terenzi, Fulvia, Merrick, William C., Sen, Ganes C.

Volltext

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells von Mingozzi, Federico, Meulenberg, Janneke J., Hui, Daniel J., Basner-Tschakarjan, Etiena, Hasbrouck, Nicole C., Edmonson, Shyrie A., Hutnick, Natalie A., Betts, Michael R., Kastelein, John J., Stroes, Erik S., High, Katherine A.

Volltext

CD8 + T-cell responses to adeno-associated virus capsid in humans von High, Katherine A, Mingozzi, Federico, Maus, Marcela V, Hui, Daniel J, Sabatino, Denise E, Murphy, Samuel L, Rasko, John E J, Ragni, Margaret V, Manno, Catherine S, Sommer, Jurg, Jiang, Haiyan, Pierce, Glenn F, Ertl, Hildegund C J

Volltext

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors von Pien, Gary C, Basner-Tschakarjan, Etiena, Hui, Daniel J, Mentlik, Ashley N, Finn, Jonathan D, Hasbrouck, Nicole C, Zhou, Shangzhen, Murphy, Samuel L, Maus, Marcela V, Mingozzi, Federico, Orange, Jordan S, High, Katherine A

Volltext

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes von Hui, Daniel J, Basner-Tschakarjan, Etiena, Chen, Yifeng, Davidson, Robert J, Buchlis, George, Yazicioglu, Mustafa, Pien, Gary C, Finn, Jonathan D, Haurigot, Virginia, Tai, Alex, Scott, David W, Cousens, Leslie P, Zhou, Shangzhen, De Groot, Anne S, Mingozzi, Federico

Volltext

Enhanced T cell function in a mouse model of human glycosylation von Buchlis, George, Odorizzi, Pamela, Soto, Paula C, Pearce, Oliver M T, Hui, Daniel J, Jordan, Martha S, Varki, Ajit, Wherry, E John, High, Katherine A

Volltext

A new pathway of translational regulation mediated by eukaryotic initiation factor 3 von Guo, Jinjiao, Hui, Daniel J., Merrick, William C., Sen, Ganes C.

Volltext

Identification of Mouse AAV Capsid-specific CD8 T Cell Epitopes von Sabatino, Denise E, Mingozzi, Federico, Hui, Daniel J, Chen, Haifeng, Colosi, Peter, Ertl, Hildegund C J, High, Katherine A

Volltext

1086. T Cell Responses to Canine Factor IX and AAV Capsid Antigens in Hemophilia B Dogs after Intravascular Gene Delivery to Skeletal Muscle von Hui, Daniel J., Mingozzi, Federico, Dillow, Aaron, McCorquodale, Stephanie, Nichols, Timothy C., Arruda, Valder R., High, Katherine A.

Volltext

Viral Stress-inducible Protein p56 Inhibits Translation by Blocking the Interaction of eIF3 with the Ternary Complex eIF2·GTP·Met-tRNAi von Hui, Daniel J., Bhasker, C.Ramana, Merrick, William C., Sen, Ganes C.

Volltext

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response von Hui, Daniel J, Liu, Yun, Patel, Ruchi, Chen, Yifeng, George, Lindsey A., Sullivan, Spencer K., Giermasz, Adam, Rasko, John, Samelson-Jones, Ben J., Ducore, Jonathan M., Dasen, Sue, Carr, Marcus E., Anguela, Xavier M., High, Katherine A.

Volltext

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds von High, Katherine A., George, Lindsey A., Eyster, M. Elaine, Sullivan, Spencer K., Ragni, Margaret V., Croteau, Stacy E., Samelson-Jones, Ben J., Evans, Matthew, Joseney-Antoine, Marcelyne, Macdougall, Amy, Kadosh, Judith, Runoski, Alexa R., Campbell-Baird, Cynthia, Douglas, Kayla, Tompkins, Summer, Hait, Howard, Couto, Linda B., Bassiri, Ashlyn Eaton, Valentino, Leonard A., Carr, Marcus E., Hui, Daniel J, Wachtel, Katie, Takefman, Daniel, Mingozzi, Federico, Anguela, Xavier M., Reape, Kathleen B

Volltext

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a von George, Lindsey A., Ragni, Margaret V., Samelson-Jones, Ben J., Cuker, Adam, Runoski, Alexa R., Cole, Grace, Wright, Fraser, Chen, Yifeng, Hui, Daniel J, Wachtel, Katie, Takefman, Daniel, Couto, Linda B., Reape, Kathleen Z., Carr, Marcus E., Anguela, Xavier M., High, Katherine A.

Volltext

185. Safety Study by Validated Immunoassays in a Phase III Study of Subjects with Inherited Retinal Dystrophy Due to Mutations in the Gene Encoding Human Retinal Pigment Epithelium... von Hui, Daniel J., Chen, Yifeng, Antrilli, Thomas, Liu, Yun, Corbau, Romuald, Russell, Stephen R., Maguire, Albert M., Bennett, Jean, High, Katherine A.

Volltext

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persi... von George, Lindsey A., Sullivan, Spencer K., Giermasz, Adam, Samelson-Jones, Ben J., Ducore, Jonathan M., Teitel, Jerome M., Cuker, Adam, Von Mackensen, Sylvia, Majumdar, Suvankar, McGuinn, Catherine E., Runoski, Alexa R., Wright, Fraser, Dasen, Sue, Barber, Kimberly, Chen, Yifeng, Hui, Daniel J., Patel, Ruchi, Liu, Yun, Wachtel, Katie, Takefman, Dan, Couto, Linda B., Reape, Kathleen Z., Carr, Marcus E., Anguela, Xavier M., High, Katherine A.

Volltext