Zobrazeno 1 - 10
of 10
pro vyhledávání: '"Ben J. Samelson-Jones"'
Autor:
Lucas Van Gorder, Bhavya S. Doshi, Elinor Willis, Matthew Lanza, Valder R. Arruda, Mary Beth Callan, Ben J. Samelson-Jones
Publikováno v:
Blood. 140:4919-4920
Autor:
Bhavya S Doshi, Ben J Samelson-Jones, Timothy C Nichols, Elizabeth P Merricks, Valder R Arruda, Mary Beth Callan
Publikováno v:
Blood. 140:7780-7781
Autor:
Kyumin Lee, Julia Q Chau, Anna R Sternberg, Allyson M Pishko, Lindsey George, Ben J Samelson-Jones
Publikováno v:
Blood. 140:5570-5571
Autor:
Robert T. N. Chen, Bhavya S. Doshi, Ben J. Samelson-Jones, Don L. Siegel, Timothy C. Nichols, Vijay Bhoj, Michael C. Milone, Valder R. Arruda
Publikováno v:
Blood. 138:3173-3173
The development of inhibitors to Factor VIII (FVIII) is a major complication of hemophilia A (HA) treatment. Most preclinical studies in HA animals have been limited to a xenoprotein response, e.g., evaluating the murine immune response to human FVII
Autor:
Jonathan M. Ducore, John E.J. Rasko, Lynne Smith, Adam Giermasz, Jerome M. Teitel, A Chhabra, Spencer K. Sullivan, Jeremy Rupon, Ben J. Samelson-Jones, Lindsey A. George, Amanda C. O’Brien, Catherine E. McGuinn, Ian Winburn
Publikováno v:
Blood. 138:3975-3975
Fifteen patients with moderately severe to severe hemophilia B (factor IX [FIX] activity ≤2%) were treated with fidanacogene elaparvovec at a dose of 5e11 vg/kg as part of a phase 1/2a study. The study was 52 weeks in duration, after which patients
Publikováno v:
Expert Opinion on Orphan Drugs. 3:997-1010
The recent success of early-phase clinical trials for adeno-associated viral (AAV) liver-directed gene therapy for hemophilia B (HB) demonstrates the potential for gene therapy, in the future, to succeed protein-based prophylaxis therapy for HB. Sign
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:
Ben J. Samelson-Jones, Syun Ru Yeh
Publikováno v:
Biochemistry. 45:8527-8538
Indoleamine 2,3-dioxygenase (IDO) is a heme-containing enzyme, which catalyzes the initial and rate-determining step of L-tryptophan (L-Trp) metabolism via the kynurenine pathway in nonhepatic tissues. Similar to inducible nitric oxide synthase (iNOS
Publikováno v:
Blood. 128:1384-1384
Recent clinical trial successes of AAV-based gene therapy for hemophilia B utilizing the hyperactive factor (F) IX variant, Padua (Arg338Leu), demonstrate the potential of this strategy to achieve disease ameliorating FIX activity levels at lower vec
Publikováno v:
Blood. 128:SCI-9
Novel approaches to enhance the biologic activity of therapeutic proteins have the potential to improve protein- and gene-based therapy for hemophilia. We have identified the variant Factor IX Padua (FIX) (R338L) with 8-fold increase in specific acti