Zobrazeno 1 - 10
of 31
pro vyhledávání: '"Daniel J Hui"'
Autor:
Daniel J Hui, Shyrie C Edmonson, Gregory M Podsakoff, Gary C Pien, Lacramioara Ivanciu, Rodney M Camire, Hildegund Ertl, Federico Mingozzi, Katherine A High, Etiena Basner-Tschakarjan
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss , Pp - (2015)
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 in humans and even market approval for a first gene therapy product in Europe. Administrati
Externí odkaz:
https://doaj.org/article/c3c459a322e749a9b9b077cc70a13749
Autor:
Xavier M. Anguela, Katie Wachtel, Judith Kadosh, Stacy E. Croteau, Marcus E. Carr, Lindsey A. George, Leonard A. Valentino, Matthew S. Evans, Federico Mingozzi, Linda B. Couto, Daniel Takefman, Kathleen B Reape, Daniel J. Hui, Alexa R. Runoski, Amy Macdougall, Katherine A. High, Cynthia Campbell-Baird, Marcelyne Joseney-Antoine, Ben J. Samelson-Jones, Howard Hait, Summer Tompkins, Spencer K. Sullivan, Ashlyn Eaton Bassiri, M. Elaine Eyster, Kayla Douglas, Margaret V. Ragni
Publikováno v:
Blood. 132:487-487
Gene transfer for hemophilia A offers the potential for a one-time disease altering treatment, eliminating the risk of bleeds while freeing patients from the burden of lifelong chronic therapy. SPK-8011 consists of a bioengineered AAV capsid expressi
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, Jurg M. Sommer, Margaret V. Ragni, Catherine S. Manno, Hildegund C.J. Ertl, Katherine A. High, Glenn Pierce, John E.J. Rasko, Haiyan Jiang, Marcela V. Maus, Daniel J. Hui, Federico Mingozzi, Samuel L. Murphy
Publikováno v:
Nature Medicine. 13:419-422
Hepatic adeno-associated virus (AAV)-serotype 2 mediated gene transfer results in transgene product expression that is sustained in experimental animals but not in human subjects. We hypothesize that this is caused by rejection of transduced hepatocy
Publikováno v:
Journal of Biological Chemistry. 281:34064-34071
Human P54 and P56 proteins are tetratricopeptide proteins that are encoded by two closely related genes, ISG54 and ISG56. These genes are induced strongly but transiently when cells are treated with interferons or double-stranded RNA or infected with
Publikováno v:
Journal of Biological Chemistry. 280:3433-3440
Members of the p56 family of mammalian proteins are strongly induced in virus-infected cells and in cells treated with interferons or double-stranded RNA. Previously, we have reported that human p56 inhibits initiation of translation by binding to th
Autor:
Jean Bennett, Yifeng Chen, Romuald Corbau, Daniel J. Hui, Stephen R. Russell, Thomas Antrilli, Yun Liu, Katherine A. High, Albert M. Maguire
Publikováno v:
Molecular Therapy. 24:S72-S73
Mutations in the gene encoding Retinal Pigment Epithelium-Specific Protein 65 (RPE65) cause impaired vision from an early age and eventually total vision loss later in life. Recent work by our group and others demonstrated the use of SPK-RPE65, an ad
Autor:
Federico Mingozzi, Daniel J. Hui, Hildegund C.J. Ertl, Shyrie C Edmonson, Rodney M. Camire, Etiena Basner-Tschakarjan, Lacramioara Ivanciu, Katherine A. High, Gregory M. Podsakoff, Gary C. Pien
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 2, Iss, Pp-(2015)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
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 in humans and even market approval for a first gene therapy product in Europe. Administrati
Publikováno v:
The EMBO Journal. 19:6891-6899
We report a new pathway of translation regulation that may operate in interferon‐treated or virus‐infected mammalian cells. This pathway is activated by P56, a protein whose synthesis is strongly induced by interferons or double‐stranded RNA. U
Autor:
Alex Tai, Federico Mingozzi, J F Wright, Carolann Howard, Daniel J. Hui, Giulia Pavani, Christian Hinderer, Yifeng Chen, Xavier M. Anguela, Gregory M. Podsakoff, Liron Elkouby, Armida Faella, Robert J. Davidson, Shangzhen Zhou, Etiena Basner-Tschakarjan, Katherine A. High, Mustafa N. Yazicioglu
Publikováno v:
Sci Transl Med
Sci Transl Med, 2013, 5 (194), pp.194ra92. ⟨10.1126/scitranslmed.3005795⟩
Sci Transl Med, 2013, 5 (194), pp.194ra92. ⟨10.1126/scitranslmed.3005795⟩
International audience; Adeno-associated virus (AAV) vectors delivered through the systemic circulation successfully transduce various target tissues in animal models. However, similar attempts in humans have been hampered by the high prevalence of n
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6c78c7b567348c1e6cc35478e67f6de4
https://hal.archives-ouvertes.fr/hal-02881152
https://hal.archives-ouvertes.fr/hal-02881152