Zobrazeno 1 - 10
of 21
pro vyhledávání: '"Gabriela Denning"'
Autor:
Athena L. Russell, Chengyu Prince, Taran S. Lundgren, Kristopher A. Knight, Gabriela Denning, Jordan S. Alexander, Jaquelyn T. Zoine, H. Trent Spencer, Shanmuganathan Chandrakasan, Christopher B. Doering
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss , Pp 710-727 (2021)
Hematopoietic stem and progenitor cell (HSPC) lentiviral gene therapy is a promising strategy toward a lifelong cure for hemophilia A (HA). The primary risks associated with this approach center on the requirement for pre-transplantation conditioning
Externí odkaz:
https://doaj.org/article/b389bd2ba16049b3ab15cefbbc027227
Autor:
Gabriela Denning
Publikováno v:
Cell and Gene Therapy Insights. 7:1153-1158
Autor:
Fania Szlam, Christopher W. Coyle, Ernest T. Parker, Gabriela Denning, Roman M. Sniecinski, Eric A. Gaucher, Christopher B. Doering, H. Trent Spencer, Chen Michelle H, Jordan M. Shields, Kristopher A. Knight, Anatolii Purchel, Andrew Fedanov, Caelan E. Radford, Pete Lollar
Publikováno v:
Blood Adv
Orthologous proteins contain sequence disparity guided by natural selection. In certain cases, species-specific protein functionality predicts pharmacological enhancement, such as greater specific activity or stability. However, immunological barrier
Autor:
Rodney M. Camire, Miguel A. Escobar, Navdeep Jhita, Michelle D. Ho, Angel A Rivera, Christopher B. Doering, Karen L. Zimowski, Christine L. Kempton, Gabriela Denning, Jordan E Shields, Julie Wechsler, Teodolinda Petrillo
Publikováno v:
Journal of Thrombosis and Haemostasis
Background Elucidating the molecular pathogenesis underlying the East Texas Bleeding Disorder (ET) led to the discovery of alternatively spliced F5 transcripts harboring large deletions within exon 13. These alternatively spliced transcripts produce
Autor:
Jordan S. Alexander, Athena Liza Russell, Kristopher A. Knight, Jaquelyn T. Zoine, Gabriela Denning, Chengyu Prince, Christopher B. Doering, Taran S. Lundgren, H. Trent Spencer, Shanmuganathan Chandrakasan
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss, Pp 710-727 (2021)
Molecular Therapy: Methods & Clinical Development, Vol 21, Iss, Pp 710-727 (2021)
Hematopoietic stem and progenitor cell (HSPC) lentiviral gene therapy is a promising strategy toward a lifelong cure for hemophilia A (HA). The primary risks associated with this approach center on the requirement for pre-transplantation conditioning
Autor:
Taran S. Lundgren, Gabriela Denning, Sean R. Stowell, H. Trent Spencer, Christopher B. Doering
Publikováno v:
Blood advances. 6(8)
Advances in the development of novel treatment options for hemophilia A are prevalent. However, the anti–factor VIII (FVIII) neutralizing antibody (inhibitor) response to existing FVIII products remains a major treatment challenge. Although some no
Autor:
Ernest T Parker, Alok Srivastava, Jordan E Shields, Gabriela Denning, Eli J Fine, H. Trent Spencer, Pete Lollar, Christopher B. Doering
Publikováno v:
Human Gene Therapy. 29:1183-1201
Genetically modified, autologous hematopoietic stem and progenitor cells (HSPCs) represent a new class of genetic medicine. Following this therapeutic paradigm, we are developing a product candidate, designated CD68-ET3-LV CD34(+), for the treatment
Autor:
Oliver Brand, Jordan E Shields, Yumiko Sakurai, Yongzhi Qiu, Joseph M. Le Doux, David R. Myers, Reginald Tran, Hommood Alrowais, Gabriela Denning, William C. Li, H. Trent Spencer, Allison M Lytle, Christopher B. Doering, Wilbur A. Lam
Publikováno v:
Molecular Therapy
Ex vivo gene therapy using lentiviral vectors (LVs) is a proven approach to treat and potentially cure many hematologic disorders and malignancies but remains stymied by cumbersome, cost-prohibitive, and scale-limited production processes that cannot
Autor:
Christopher W. Coyle, P. C. Spiegel, Pete Lollar, Anne E. d’Aquino, Andrew Fedanov, Ernest T. Parker, Christopher B. Doering, Harold Trent Spencer, Ian W. Smith, Gabriela Denning
Publikováno v:
J Thromb Haemost
BACKGROUND: Coagulation factor VIII represents one of the oldest protein-based therapeutics, serving as an effective hemophilia A treatment for half a century. Optimal treatment consists of repeated intravenous infusions of blood coagulation factor V
Autor:
Robert Moot, Christopher B. Doering, Jennifer M. Johnston, D Whitehead, Gabriela Denning, Harold Trent Spencer, J M Le Doux, Jordan E Shields
Publikováno v:
Gene Therapy. 21:1008-1020
A difficulty in the field of gene therapy is the need to increase the susceptibility of hematopoietic stem cells (HSCs) to ex vivo genetic manipulation. To overcome this obstacle a high-throughput screen was performed to identify compounds that could