Zobrazeno 1 - 10
of 15
pro vyhledávání: '"Shyrie Edmonson"'
Autor:
Katherine A. High, Virginia Haurigot, F Mingozzi, Fatima Bosch, Eduard Ayuso, Xavier M. Anguela, L Africa, Shangzhen Zhou, Shyrie Edmonson, J F Wright, Joel Montane, Xavier León
Publikováno v:
Gene Therapy. 17:503-510
The purity of adeno-associated virus (AAV) vector preparations has important implications for both safety and efficacy of clinical gene transfer. Early-stage screening of candidates for AAV-based therapeutics ideally requires a purification method th
Publikováno v:
Molecular Therapy. 16(12):1960-1967
We set out to analyze the fundamental biological differences between AAV2 and AAV8 that may contribute to their different performances in vivo. High-throughput protein interaction screens were used to identify binding partners for each serotype. Of t
Autor:
Shyrie Edmonson, Denise E. Sabatino, Katherine A. High, Hojun Li, Federico Mingozzi, Samuel L. Murphy, Daniel J. Hui
Publikováno v:
Journal of Medical Virology. 81:65-74
Humoral immune responses occur following exposure to Adeno-associated virus (AAV) or AAV vectors. Many studies characterized antibody responses to AAV, but human IgG subclass responses to AAV have not been previously described. In this study, IgG sub
Autor:
Denise E. Sabatino, Cesare Campagnoli, Tippi C. MacKenzie, Yi-Lin Liu, William H. Peranteau, Shyrie Edmonson, Alan W. Flake, Katherine A. High
Publikováno v:
Molecular Therapy. 15:1677-1685
The major complication associated with protein replacement therapy currently used in the treatment of hemophilia B (HB) is the development of antibodies to the infused human Factor IX (hF.IX). We hypothesized that vector-mediated expression of hF.IX,
Autor:
Samuel L. Murphy, Katherine A. High, Wynetta Giles-Davis, Hua Li, Shyrie Edmonson, Hildegund C.J. Ertl, Marcio O. Lasaro, Yan Li, Zhiquan Xiang
Publikováno v:
Molecular Therapy. 15:792-800
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 experimental animals. Mice immunized with adenoviral vectors expressing AAV capsid or with
Autor:
Yi Zhang, Alexander Schlachterman, Jianxiang Zou, Yi-Lin Liu, Valder R. Arruda, Joerg Schuettrumpf, Shyrie Edmonson, Weidong Xiao
Publikováno v:
Molecular Therapy. 13:88-97
Identifying factors that influence gene transfer efficacy is critical for a successful gene-based clinical study. Here we demonstrate that in vivo AAV-2-mediated gene transfer is efficiently inhibited by unfractionated heparin, but not by a heparin p
Autor:
Marc Pleimes, Shyrie Edmonson, Valder R. Arruda, Elina Armstrong, Denise E. Sabatino, Roland W. Herzog, Julie C. Fitzgerald, Yi-Lin Liu, Joerg Schuettrumpf, Katherine A. High
Publikováno v:
Blood. 104:2767-2774
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 all due to gene deletions, a type of mutation that is relatively rare in the human hemophi
Autor:
John J.P. Kastelein, Erik S.G. Stroes, Katherine A. High, Etiena Basner-Tschakarjan, Natalie A. Hutnick, Nicole C. Hasbrouck, Federico Mingozzi, Daniel J. Hui, Shyrie Edmonson, Janneke J. Meulenberg, Michael R. Betts
Publikováno v:
Blood, 114(10), 2077-2086. American Society of Hematology
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 high-dose cohort experienced a transient increase in the muscle enzyme creatine phosphoki
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ad24a6f6b59148987b7387edb309fa6f
https://pure.amc.nl/en/publications/aav1mediated-gene-transfer-to-skeletal-muscle-in-humans-results-in-dosedependent-activation-of-capsidspecific-t-cells(cccc3c39-ec78-468c-acf2-a0ef66b68c34).html
https://pure.amc.nl/en/publications/aav1mediated-gene-transfer-to-skeletal-muscle-in-humans-results-in-dosedependent-activation-of-capsidspecific-t-cells(cccc3c39-ec78-468c-acf2-a0ef66b68c34).html
Autor:
Daniel J. Hui, Glenn F. Pierce, Shangzhen Zhou, J. Fraser Wright, Federico Mingozzi, Denise E. Sabatino, Haiyan Jiang, Shyrie Edmonson, Nicole C. Hasbrouck, Katherine A. High, Etiena Basner-Tschakarjan, Valder R. Arruda
Publikováno v:
Blood. 110(7)
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 only short-term expression in humans. Loss of F.IX expression is likely due to a cytotoxi
Publikováno v:
Molecular Therapy. 13:S31-S32
Adeno-associated virus (AAV) vectors hold the potential to treat a variety of inherited and acquired human diseases. One of the confounding issues regarding the use of AAV in humans is the cellular immune response to the virus vector. While the abili