Zobrazeno 1 - 7
of 7
pro vyhledávání: '"Krystal Sandza"'
Autor:
Brian R. Long, Krystal Sandza, Jennifer Holcomb, Lucy Crockett, Gregory M. Hayes, Jeremy Arens, Carlos Fonck, Laurie S. Tsuruda, Becky Schweighardt, Charles A. O’Neill, Stephen Zoog, Christian Vettermann
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss , Pp 440-452 (2019)
Adeno-associated virus (AAV)-based vectors are widely used for gene therapy, but the effect of pre-existing antibodies resulting from exposure to wild-type AAV is unclear. In addition, other poorly defined plasma factors could inhibit AAV vector tran
Externí odkaz:
https://doaj.org/article/6c5d4d023a7e43a5aab2e49899b42daf
Ultra-sensitive AAV capsid detection by immunocapture-based qPCR following factor VIII gene transfer
Autor:
Fan Yang, Wing Yen Wong, Stephen J. Zoog, Hassibullah Akeefe, Elli Koziol, Christian Vettermann, Jennifer Holcomb, Annie Clark, Kevin Hammon, Joshua Henshaw, Nina Mitchell, Kathryn Patton, Krystal Sandza, Benjamin Kim
Publikováno v:
Gene Therapy. 29:94-105
Adeno-associated virus (AAV)-based gene therapy vectors are replication-incompetent and thus pose minimal risk for horizontal transmission or release into the environment. In studies with AAV5-FVIII-SQ (valoctocogene roxaparvovec), an investigational
Autor:
Jennifer Holcomb, Christian Vettermann, Charles A. O'Neill, Krystal Sandza, Carlos Fonck, Gregory M. Hayes, Brian Long, Lucy Crockett, Stephen J. Zoog, Jeremy Arens, Becky Schweighardt, Laurie S. Tsuruda
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 13, Iss, Pp 440-452 (2019)
Molecular Therapy. Methods & Clinical Development
Molecular Therapy. Methods & Clinical Development
Adeno-associated virus (AAV)-based vectors are widely used for gene therapy, but the effect of pre-existing antibodies resulting from exposure to wild-type AAV is unclear. In addition, other poorly defined plasma factors could inhibit AAV vector tran
Autor:
Krystal Sandza, S Triffault, Laurie S. Tsuruda, Barrie Carter, Stephen J. Zoog, Lillian E. Falese, S Gangar, Christian Vettermann, Brian Long, Bridget Yates, Sylvia Fong
Publikováno v:
Gene Therapy
Gene therapy may offer a new treatment option, particularly for patients with severe hemophilia, based on recent research. However, individuals with pre-existing immunity to adeno-associated viruses (AAVs) may be less likely to benefit from AAV vecto
Autor:
Kevin Hammon, Benjamin Kim, Chris B. Russell, Annie Clark, Joshua Henshaw, Jennifer Holcomb, Kala Jayaram, Elli Koziol, Krystal Sandza, Christian Vettermann, Richard Torres
Publikováno v:
Blood. 136:21-21
Introduction: Long-term durable expression of hFVIII-SQ has been observed following BMN 270 (AAV5-hFVIII-SQ, valoctocogene roxaparvovec) single-dose administration in patients with severe hemophilia A. Although adeno-associated virus (AAV) vectors ar
Autor:
Sherry Bullens, Charles A. O'Neill, Shinong Long, Gordon Vehar, Anil Bagri, Stuart Bunting, Nicole Galicia, Rajeev Mahimkar, Choong-Ryoul Sihn, Danielle Harmon, Laurie S. Tsuruda, Krystal Sandza, Lening Zhang, Bridget Yates, Sylvia Fong, Peter Colosi, Britta Handyside, Barrie Carter, Lin Xie
Publikováno v:
Molecular Therapy
Hemophilia A is an X-linked bleeding disorder caused by mutations in the gene encoding the factor VIII (FVIII) coagulation protein. Bleeding episodes in patients are reduced by prophylactic therapy or treated acutely using recombinant or plasma-deriv
Autor:
Shinong Long, Lin Xie, Barrie Carter, Lening Zhang, Stuart Bunting, Rajeev Mahimkar, Gordon Vehar, Sherry Bullens, Krystal Sandza, Sylvia Fong, Peter Colosi
Publikováno v:
Blood. 126:3239-3239
Hemophilia A is an X-linked bleeding disorder caused by mutations in the gene encoding the Factor VIII coagulation protein (FVIII). Bleeding episodes in patients are reduced by prophylactic therapy or treated acutely using recombinant or plasma deriv