Adeno-associated virus gene therapy for hemophilia
BJ Samelson-Jones, LA George - Annual review of medicine, 2023 - annualreviews.org
In vivo gene therapy is rapidly emerging as a new therapeutic paradigm for monogenic
disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served …
disorders. For almost three decades, hemophilia A (HA) and hemophilia B (HB) have served …
[HTML][HTML] Multiyear factor VIII expression after AAV gene transfer for hemophilia A
LA George, PE Monahan, ME Eyster… - … England Journal of …, 2021 - Mass Medical Soc
Background The goal of gene therapy for patients with hemophilia A is to safely impart long-
term stable factor VIII expression that predictably ameliorates bleeding with the use of the …
term stable factor VIII expression that predictably ameliorates bleeding with the use of the …
[HTML][HTML] Deciphering conundrums of adeno-associated virus liver-directed gene therapy: focus on hemophilia
Adeno-associated virus gene therapy has been the subject of intensive investigation for
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …
monogenic disease gene addition therapy for more than 25 years, yet few therapies have …
Roctavian gene therapy for hemophilia A
BJ Samelson-Jones, JC Small, LA George - Blood Advances, 2024 - ashpublications.org
After successful efforts in adeno-associated virus (AAV) gene addition for hemophilia B gene
therapy, the development of valoctocogene roxaparvovec (Roctavian; Biomarin) over the …
therapy, the development of valoctocogene roxaparvovec (Roctavian; Biomarin) over the …
Factor VIII trafficking to CD4+ T cells shapes its immunogenicity and requires several types of antigen-presenting cells
Despite> 80 years of clinical experience with coagulation factor VIII (FVIII) inhibitors,
surprisingly little is known about the in vivo mechanism of this most serious complication of …
surprisingly little is known about the in vivo mechanism of this most serious complication of …
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 …
[HTML][HTML] Treatment of congenital coagulopathies, from biologic to biotechnological drugs: The relevance of gene editing (CRISPR/Cas)
Congenital coagulopathies have, throughout the history of medicine, been a focus of
scientific study and of great interest as they constitute an alteration of one of the most …
scientific study and of great interest as they constitute an alteration of one of the most …
The protein C pathways
The protein C pathways : Current Opinion in Hematology The protein C pathways : Current
Opinion in Hematology Log in or Register Subscribe to journalSubscribe Get new issue alertsGet …
Opinion in Hematology Log in or Register Subscribe to journalSubscribe Get new issue alertsGet …
Thrombin spatial distribution determines protein C activation during hemostasis and thrombosis
Rebalancing the hemostatic system by targeting endogenous anticoagulant pathways, like
the protein C (PC) system, is being tested as a means of improving hemostasis in patients …
the protein C (PC) system, is being tested as a means of improving hemostasis in patients …
Factor VIII mutated with Lys1813Ala within the factor IXa-binding region enhances intrinsic coagulation potential
Y Nakajima, M Takeyama, A Oda… - Blood …, 2023 - ashpublications.org
Factor VIII (FVIII) functions as a cofactor of FIXa for FX activation in the intrinsic tenase
complex. The 1811-1818 region in the FVIII A3 domain was observed to contribute to FIXa …
complex. The 1811-1818 region in the FVIII A3 domain was observed to contribute to FIXa …