Zobrazeno 1 - 10
of 50
pro vyhledávání: '"Linda B. Couto"'
Autor:
Johannes C.M. Van der Loo, Jerome M. Teitel, Benjamin J. Samelson-Jones, Adam Cuker, Xavier M. Anguela, Marcus E. Carr, Catherine E. McGuinn, Yifeng Chen, Linda B. Couto, Adam Giermasz, Suvankar Majumdar, Katie Wachtel, Jonathan M. Ducore, Stefan Tiefenbacher, Margaret V. Ragni, J. Fraser Wright, Olga Zelenaia, Katherine A. High, Yun Liu, John E.J. Rasko, Daniel Takefman, Lisa M. Sullivan, Angela Winters, Daniel Hui, Alvin Luk, Spencer K. Sullivan, Valder R. Arruda, Lindsey A. George
Publikováno v:
The New England journal of medicine. 377(23)
The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia.We infused a si
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
Publikováno v:
Molecular Therapy. 21:588-601
We developed a strategy to treat hepatitis C virus (HCV) infection by replacing five endogenous microRNA (miRNA) sequences of a natural miRNA cluster (miR-17–92) with sequences that are complementary to the HCV genome. This miRNA cluster (HCV-miR-C
Publikováno v:
Journal of Virological Methods. 173:99-107
Column chromatography has been described for purification of recombinant adeno-associated viral vectors (rAAV) serotypes 1, 2, 5, 6 and 8. Some of these purification processes have been used in manufacturing pre-clinical grade and clinical grade rAAV
Publikováno v:
Hepatology. 52:1877-1887
RNA interference (RNAi) is being evaluated as an alternative therapeutic strategy for hepatitis C virus (HCV) infection. The use of viral vectors encoding short hairpin RNAs (shRNAs) has been the most common strategy employed to provide sustained exp
Autor:
Dea Nagy, Glenn Pierce, Joseph A. Vargas, Tongyao Liu, Ciaran Scallan, Jurg M. Sommer, Haiyan Jiang, Sharmila Vijay, Federico Mingozzi, Linda B. Couto, Shangzhen Zhou, Katherine A. High, Susannah Patarroyo-White
Publikováno v:
Blood. 108:3321-3328
In a clinical study of recombinant adeno-associated virus-2 expressing human factor IX (AAV2-FIX), we detected 2 impediments to long-term gene transfer. First, preexisting anti-AAV neutralizing antibodies (NABs) prevent vector from reaching the targe
Autor:
Linda B. Couto
Publikováno v:
Seminars in Thrombosis and Hemostasis. 30:161-171
Gene therapy offers a potential cure for hemophilia and several gene transfer vectors have been evaluated for their ability to treat this disease. This article reviews the studies that have been performed to evaluate the ability of recombinant adeno-
Publikováno v:
Human Gene Therapy. 15:287-291
In a clinical safety trial involving an adeno-associated virus (AAV) gene therapy vector encoding human factor IX, intrahepatic administration of the vector was associated with the finding of vector DNA in semen that persisted for several weeks. Unce
Autor:
Joseph Vargas, Dea Nagy, Haig H. Kazazian, Sharon K. Powell, Amy E. Parker, Tongyao Liu, Haifeng Chen, J. Fraser Wright, Linda B. Couto, Rita Sarkar, Susannah L. Patarroyo-White, Ciaran Scallan, Alan McClelland, Haiyan Jiang
Publikováno v:
Blood. 102:3919-3926
Using separate adeno-associated viral 2 (AAV2) vectors to deliver the heavy and light chains of factor VIII (FVIII) we have overcome the packaging limitations of AAV, achieving phenotypic correction of hemophilia A in mice. AAV vectors were construct
Autor:
Katie Wachtel, Marcus E. Carr, Katherine A. High, Lisa M. Sullivan, Spencer K. Sullivan, Adam Giermasz, Yifeng Chen, Aline M. Galvão, Linda B. Couto, Alvin Luk, J. Fraser Wright, Jerome M. Teitel, Jonathan M. Ducore, Daniel J. Hui, Adam Cuker, Xavier M. Anguela, Catherine E. McGuinn, Suvankar Majumdar, Daniel Takefman, Lindsey A. George, Teresa Urich
Publikováno v:
Blood. 128:3-3
Background: Earlier data demonstrated long-term expression of factor IX (mean FIX:C ~5.1%) following AAV8-mediated gene transfer at 2 x1012 vg/kg in hemophilia B (Nathwani et al., 2014). While the clinical improvement imparted by stable FIX levels is