[HTML][HTML] IL-15 blockade and rapamycin rescue multifactorial loss of factor VIII from AAV-transduced hepatocytes in hemophilia A mice
JSS Butterfield, K Yamada, TB Bertolini, F Syed… - Molecular Therapy, 2022 - cell.com
Hepatic adeno-associated viral (AAV) gene transfer has the potential to cure the X-linked
bleeding disorder hemophilia A. However, declining therapeutic coagulation factor VIII …
bleeding disorder hemophilia A. However, declining therapeutic coagulation factor VIII …
Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human factor IX variant in mice and rhesus macaques
K Pekrun, CJ Stephens, A Gonzalez-Sandoval… - Molecular Therapy, 2024 - cell.com
We investigated long-term human coagulation factor IX (huFIX) expression of a novel variant
when delivered into mice and rhesus macaques and compared transduction efficiencies …
when delivered into mice and rhesus macaques and compared transduction efficiencies …
[HTML][HTML] Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice
AR Sternberg, C Martos-Rus, RJ Davidson… - Nature …, 2024 - nature.com
Durable factor VIII expression that normalizes hemostasis is an unrealized goal of
hemophilia A adeno-associated virus-mediated gene therapy. Trials with initially normal …
hemophilia A adeno-associated virus-mediated gene therapy. Trials with initially normal …
First-in-patient dose prediction for adeno-associated virus-mediated hemophilia gene therapy using allometric scaling
P Zou - Molecular Pharmaceutics, 2022 - ACS Publications
In this study, the author compared the performance of two allometric scaling approaches and
body-weight-based dose conversion approach for first-in-patient (FIP) dose prediction for …
body-weight-based dose conversion approach for first-in-patient (FIP) dose prediction for …
Coagulation factor VIII regulates von Willebrand factor homeostasis in vivo
W Cao, AR Trask, AI Bignotti, LA George… - Journal of Thrombosis …, 2023 - Elsevier
Abstract Background Coagulation factor VIII (FVIII) and von Willebrand factor (VWF) circulate
as a noncovalent complex, but each has its distinct functions. Binding of FVIII to VWF results …
as a noncovalent complex, but each has its distinct functions. Binding of FVIII to VWF results …
[HTML][HTML] Altered cleavage of human factor VIII at the B-domain and acidic region 3 interface enhances expression after gene therapy in hemophilia A mice
GN Nguyen, JR Lindgren, MC Seleme, S Kafle… - Journal of Thrombosis …, 2023 - Elsevier
Background Variants of human factor VIII (hFVIII) have been developed to further
understand the structure and function of hFVIII and improve gene-based therapeutics. We …
understand the structure and function of hFVIII and improve gene-based therapeutics. We …
[HTML][HTML] Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy
RA Potter, DA Griffin, KN Heller, JR Mendell… - Biology …, 2023 - journals.biologists.com
Robust expression of shortened, functional dystrophin provided impetus to develop adeno-
associated virus (AAV)–based constructs for clinical application. Because several cassettes …
associated virus (AAV)–based constructs for clinical application. Because several cassettes …
Pharmacokinetic analysis identifies a factor VIII immunogenicity threshold after AAV gene therapy in hemophilia A mice
TS Lundgren, G Denning, SR Stowell… - Blood …, 2022 - ashpublications.org
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 …
However, the anti–factor VIII (FVIII) neutralizing antibody (inhibitor) response to existing FVIII …