Zobrazeno 1 - 6
of 6
pro vyhledávání: '"Katie Wachtel"'
Autor:
Margareth C. Ozelo, Katie Wachtel, Yifeng Chen, Benjamin J. Samelson-Jones, Leslie Raffini, Katherine A. High, Margaret V. Ragni, John E.J. Rasko, Xavier M. Anguela, Lindsey A. George, Jennifer Wellman, Alexa R. Runowski, Federico Mingozzi, Maria Hazbon, Klaudia Kuranda
Publikováno v:
Mol Ther
Adeno-associated virus (AAV) vectors are a leading platform for gene-based therapies for both monogenic and complex acquired disorders. The success of AAV gene transfer highlights the need to answer outstanding clinical questions of safety, durabilit
Autor:
Xiaosong Zhu, Jean Bennett, Chris A. Johnson, Katie Wachtel, Jean Walshire, Zi Fan Yu, Julie Pappas, Taylor Kehoe, Elliott H. Sohn, Jennifer Wellman, Vinit B. Mahajan, Bart P. Leroy, Michelle T. Weckmann, Francesca Simonelli, Okan U. Elci, Dominique Cross, Amy Tillman, J. Fraser Wright, Lindsey A. George, Kathleen A. Marshall, Julia A. Haller, Maria C. Davis, Edwin M. Stone, Janet Wittes, Laura E. Dingfield, Dina Y. Gewaily, Sarah McCague, Leslie Raffini, Katherine A. High, Albert M. Maguire, Hannah Reichert, Arlene V. Drack, Daniel C. Chung, Wanda Pfeifer, F. Parker Hudson, Stephen R. Russell
Publikováno v:
The Lancet. 390:849-860
Background Phase 1 studies have shown potential benefit of gene replacement in RPE65-mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of voretigene neparvovec in participants whose inherited retinal dystrophy
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
Autor:
Andres Castillo, Deborah Moorad-Doctor, Ruthie H Ratcliffe, Gregory E. Garcia, Harry Singh, Katie Wachtel
Publikováno v:
Journal of Analytical Toxicology. 31:69-74
A rapid and sensitive assay for pyridinium oximes in plasma and tissue was developed. The method was suitable for the analysis of mono- and di-pyridinium oximes and utilizes ultrafiltration followed by cation-exchange high-performance liquid chromato
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