Search Results - "Edmonson, Shyrie"

Assessing the potential for AAV vector genotoxicity in a murine model by Li, Hojun, Malani, Nirav, Hamilton, Shari R., Schlachterman, Alexander, Bussadori, Giulio, Edmonson, Shyrie E., Shah, Rachel, Arruda, Valder R., Mingozzi, Federico, Fraser Wright, J., Bushman, Frederic D., High, Katherine A.

“…Gene transfer using adeno-associated virus (AAV) vectors has great potential for treating human disease. Recently, questions have arisen about the safety of…”
Get full text

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells by 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.

“…In a clinical trial for adeno-associated virus serotype 1 (AAV-1)–mediated gene transfer to muscle for lipoprotein lipase (LPL) deficiency, 1 subject from the…”
Get full text

Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B by Mingozzi, Federico, Chen, Yifeng, Murphy, Samuel L, Edmonson, Shyrie C, Tai, Alex, Price, Sandra D, Metzger, Mark E, Zhou, Shangzhen, Wright, J Fraser, Donahue, Robert E, Dunbar, Cynthia E, High, Katherine A

“…Liver gene transfer for hemophilia B has shown very promising results in recent clinical studies. A potential complication of gene-based treatments for…”
Get full text

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver by 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.

“…Adeno-associated virus (AAV)–mediated gene transfer of factor IX (F.IX) to the liver results in long-term expression of transgene in experimental animals, but…”
Get full text

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

“…Humoral immune responses occur following exposure to Adeno-associated virus (AAV) or AAV vectors. Many studies characterized antibody responses to AAV, but…”
Get full text

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes by 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

“…Adeno-associated virus (AAV) has become one of the most promising vectors in gene transfer in the last 10 years with successful translation to clinical trials…”
Get full text

Persistent Expression of hF.IX After Tolerance Induction by In Utero or Neonatal Administration of AAV-1-F.IX in Hemophilia B Mice by Sabatino, Denise E, MacKenzie, Tippi C, Peranteau, William, Edmonson, Shyrie, Campagnoli, Cesare, Liu, Yi-Lin, Flake, Alan W, High, Katherine A

“…The major complication associated with protein replacement therapy currently used in the treatment of hemophilia B (HB) is the development of antibodies to the…”
Get full text

Pre-existing AAV Capsid-specific CD8+ T Cells are Unable to Eliminate AAV-transduced Hepatocytes by Li, Hua, Murphy, Samuel L, Giles-Davis, Wynetta, Edmonson, Shyrie, Xiang, Zhiquan, Li, Yan, Lasaro, Marcio O, High, Katherine A, Ertl, Hildegund CJ

“…The goal of these studies was to test whether adeno-associated virus (AAV) capsid-specific CD8+ T cells cause loss of hepatic AAV-mediated gene expression in…”
Get full text

High-throughput Screening and Biophysical Interrogation of Hepatotropic AAV by Murphy, Samuel L, Bhagwat, Anand, Edmonson, Shyrie, Zhou, Shangzhen, High, Katherine A

“…We set out to analyze the fundamental biological differences between AAV2 and AAV8 that may contribute to their different performances in vivo. High-throughput…”
Get full text

Assessment of Insertional Mutagenesis Risk Following AAV Vector-Mediated Factor IX Gene Transfer in Mice by Li, Hojun, Malani, Nirav, Hamilton, Shari R, Schlachterman, Alexander, Zhang, Jim Z, Bussadori, Giulio, Edmonson, Shyrie A, Mingozzi, Federico, Wright, J. Fraser, Arruda, Valder R., Bushman, Frederic D, High, Katherine A.

“…Abstract 2465 Poster Board II-442 Gene therapy for Hemophilia B is a promising alternative to recombinant protein therapy. Long-term expression of coagulation…”
Get full text

Immunosuppression Modulates Immune Responses to AAV Capsid in Human Subjects Undergoing Intramuscular Gene Transfer for Lipoprotein Lipase Deficiency by Hui, Daniel J, Mingozzi, Federico, Kleefstra, Annemarie, Meulenberg, Janneke M, Edmonson, Shyrie, Morin, Denise, Gaudet, Daniel, van Deventer, Sander, High, Katherine A.

“…Administration of adeno-associated viral vectors (AAV) has resulted in long-term therapeutic gene transfer in multiple large animal models of disease, but…”
Get full text

Conserved Amino Acid Sequences in Parvovirus B19 and Adeno-Associated Virus Stimulate Functionally Cross-Reactive CD8 T Cells by Murphy, Samuel L., Hui, Daniel J., Edmonson, Shyrie, High, Katherine A.

“…Use of adeno-associated virus (AAV) to achieve liver-targeted gene transfer for the treatment of hemophilia B in human subjects resulted in the expression of…”
Get full text

74. Cellular Immunity to Adeno-Associated Virus Capsid Attenuates Transgene Expression in the Liver by Murphy, Samuel L., Sabatino, Denise, Mingozzi, Federico, Edmonson, Shyrie, High, Katherine

“…Adeno-associated virus (AAV) vectors hold the potential to treat a variety of inherited and acquired human diseases. One of the confounding issues regarding…”
Get full text

Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene by Sabatino, Denise E., Armstrong, Elina, Edmonson, Shyrie, Liu, Yi-Lin, Pleimes, Marc, Schuettrumpf, Joerg, Fitzgerald, Julie, Herzog, Roland W., Arruda, Valder R., High, Katherine A.

“…Animal models have been critical to the development of novel therapeutics in hemophilia. A deficiency of current murine models of hemophilia B is that they are…”
Get full text

The inhibitory effects of anticoagulation on in vivo gene transfer by adeno-associated viral or adenoviral vectors by Schuettrumpf, Joerg, Zou, Jianxiang, Zhang, Yi, Schlachterman, Alexander, Liu, Yi-Lin, Edmonson, Shyrie, Xiao, Weidong, Arruda, Valder R

“…Identifying factors that influence gene transfer efficacy is critical for a successful gene-based clinical study. Here we demonstrate that in vivo…”
Get full text

A Novel Role of Coagulation Proteases on Viral-Based Gene Transfer Efficacy by Schuettrumpf, Joerg, Zou, Jianxiang, Tai, Shin Jen, Schlachterman, Alexander, Tian, Kian, Edmonson, Shyrie, Liu, Jianhua, Andrade-Gordon, Patricia, High, Katherine, Arruda, Valder

“…Coagulation proteases are crucial for hemostasis and have also been implicated in inflammatory responses, blood vessel formation, and tumor cell metastasis…”
Get full text

37. Duration of Immunologically Detectable AAV-2 Capsid in an Animal Model by Sabatino, Denise E, Mingozzi, Federico, Brooks, David C, Schlachterman, Alex, Edmonson, Shyrie, Couto, Linda, Colosi, Peter, Chen, Haifeng, Ertl, Hildegund C J, High, Katherine A

“…In a Phase I trial for Hemophilia B six subjects underwent infusion of an adeno-associated viral (AAV) vector expressing human Factor IX (F.IX) under the…”
Get full text